Rare disease initiative aims to speed diagnoses and treatment in Latin America
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article |
2024/11/01 |
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Improving the recipe for gene therapy: New treatment for Gaucher disease shows promise
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article |
2024/10/18 |
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Family selling dream home to fund life-saving treatment for 5-year-old daughter
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article |
2024/10/18 |
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Impossible' FDA adcomm ends in favor of Stealth’s ultra-rare disease med
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article |
2024/10/12 |
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Sentynl Therapeutics: Next Generation Sequencing (NGS) and Rare Disease Diagnostics
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article |
2024/10/12 |
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TikTok star Taylor Rousseau Grigg died after complications from Addison's disease, family says. What to know about the rare condition.
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article |
2024/10/12 |
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7-Year-Old with Rare Disease — 'Basically Childhood Dementia' — Needs $172,000 Therapy Every 2 Weeks
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article |
2024/10/12 |
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Our Rare Disease Network Goes Global
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article |
2024/10/04 |
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The Future of Rare Disease Treatment with Precision Medicine
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article |
2024/10/04 |
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RARE's Wilson Disease Candidate Betters Standard Therapy in Study
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article |
2024/10/04 |
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NHS launches study of newborn blood test to detect 200 rare conditions
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article |
2024/10/04 |
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Why your chance of having a rare disease depends on where you live
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article |
2024/10/04 |
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Sanofi Drug for Rare Blood Disease Is Heading to Recordati for $825M
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article |
2024/10/04 |
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Using AI to repurpose existing drugs for treatment of rare diseases
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article |
2024/09/27 |
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Scientists Discover Gene Responsible for Rare, Inherited Eye Disease, NIH Reports
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article |
2024/09/27 |
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Rare Diagnoses Change People’s Perception of Medical Risk
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article |
2024/09/27 |
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I knew something was wrong with my newborn but doctors ignored me- months later my baby was diagnosed with a rare disease
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article |
2024/09/27 |
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7-year-old girl with rare disease forced to hide from sun, cover every inch of skin: ‘Complete gut punch’
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article |
2024/09/27 |
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Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
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article |
2024/09/20 |
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US FDA approves Zevra's treatment for rare genetic disease
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article |
2024/09/20 |
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Rare eye disease doesn't stop sports photographer from focusing on his dream
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article |
2024/09/13 |
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‘My Low Back Pain Turned Out To Be A Rare Inflammatory Disease With No Cure’
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article |
2024/09/13 |
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There Was No Treatment for His Son’s Rare Disease, so Dad Moves Mountains to Make One for Children Worldwide
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article |
2024/09/06 |
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Beyond Rare Disease: Wet AMD Gene Therapy Could Reach Millions
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article |
2024/09/06 |
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RARE Navigator Pilot Program Aims to Eliminate Critical Gaps in Support for Patients and Families Newly Diagnosed with a Rare Disease
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article |
2024/09/06 |
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EU Court halts Commission’s ban on drug for rare liver disease
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article |
2024/09/06 |
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Groundbreaking Study by RDDC and NORD: Unveiling Inequities in the Rare Disease Community Through the Voices of Diverse Patients and Caregivers
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article |
2024/08/29 |
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The most expensive drugs in the US
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article |
2024/08/29 |
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EEE virus: What is the mosquito-borne disease affecting the US and how deadly is it?
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article |
2024/08/29 |
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Rare diseases point to connections between metabolism and immunity
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article |
2024/08/24 |
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What Is Angelman Syndrome? Everything to Know About the Rare Genetic Disorder Colin Farrell’s Son James Has
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article |
2024/08/10 |
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After 40 years of pain, Erie man diagnosed with life-changing rare disease
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article |
2024/08/10 |
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Rare Disease-Focused Applied Therapeutics' Lead Candidate Govorestat Has Blockbuster Potential, Analyst Sees 140% Stock Upside
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article |
2024/08/03 |
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‘This is real’: Newport Beach family seeks to ensure rare disease funding continues
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article |
2024/08/03 |
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Genetic diagnostics of ultra-rare diseases: Large multicenter study identifies 34 new genetic diseases
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article |
2024/07/27 |
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Inside the efforts to rescue a rare disease gene therapy
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article |
2024/07/27 |
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My Uncle Donald Trump Told Me Disabled Americans Like My Son ‘Should Just Die’
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article |
2024/07/27 |
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Using AI to Improve Detection of Rare Diseases
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article |
2024/07/19 |
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Ionis (IONS) Completes Enrolment for Rare Disease Drug Study
|
article |
2024/07/19 |
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FDA launches new rare disease innovation hub
|
article |
2024/07/19 |
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‘Crouching Tiger, Hidden Dragon’ star Cheng Pei-pei dead at 78
|
article |
2024/07/19 |
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Connecting the Clinical, Regulatory, and Commercial Dots for Rare Diseases
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article |
2024/07/12 |
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What is ALS? Oak Ridge Boys member Joe Bonsall died from complications from this rare disease. Here's what to know
|
article |
2024/07/12 |
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AI-Powered Tool Combs through Electronic Health Records For Faster Diagnoses of Rare Diseases
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article |
2024/07/06 |
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Left in limbo: When pharma halts rare disease research
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article |
2024/07/06 |
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Gene therapy halts progression of rare genetic condition in young boy
|
article |
2024/06/28 |
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‘There is no cure for this’: Rare disease causing 6-year-old girl to age rapidly
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video |
2024/06/28 |
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AWS commits $10m for paediatric health and rare diseases research
|
article |
2024/06/28 |
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FDA Opens Doors for More Treatments for Rare Diseases through the New START Pilot Program
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article |
2024/06/28 |
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Making genome sequencing a first-line test in rare disease
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article |
2024/06/28 |
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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
|
article |
2024/06/23 |
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Whole Genome Sequencing Proven Superior for Identifying Genetic Causes of Rare Diseases
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article |
2024/06/15 |
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Vertex says Casgevy benefit extends to nearly five years in sickle cell disease
|
article |
2024/06/15 |
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Beyond Biotech - the podcast from Labiotech Developing treatments for rare pediatric diseases
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podcast |
2024/06/08 |
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Moderna and Myrtelle among those picked for FDA rare disease programme
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article |
2024/06/08 |
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Denali, Neurogene and more picked by FDA's 'Operation Warp Speed' for rare disease
|
article |
2024/06/08 |
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Moderna's MMA candidate nabs spot in FDA pilot for rare disease drugs
|
article |
2024/06/08 |
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Rare disease's mutation could explain more common conditions
|
article |
2024/06/08 |
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FDA Approves Eculizumab-Aeeb as Interchangeable Biosimilar for 2 Rare Diseases
|
article |
2024/06/02 |
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Existing drug shows promise as treatment for rare genetic disorder
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article |
2024/06/02 |
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Genetic diagnostic technology a game changer for rare diseases, but ethics concerns linger
|
article |
2024/05/27 |
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Rare disease clinical trials
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article |
2024/05/27 |
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Deadly brain disease ‘doesn’t feel rare’ to this Michigan woman’s family
|
article |
2024/05/27 |
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Peter Alexander’s Sister Rebecca Recalls Progressive Vision and Hearing Loss Since Age 12 Due to Rare Disease
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article |
2024/05/18 |
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Alabama will screen newborns for more rare diseases thanks to Foley teen, family and advocates
|
article |
2024/05/18 |
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These congressmen want to modify the Inflation Reduction Act to foster rare disease research
|
article |
2024/05/10 |
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Jaguar Health Announces Submission of Clinical Trial Applications for Crofelemer for the Rare Disease Indications Microvillus Inclusion Disease (MVID) and Short Bowel Syndrome (SBS) in Europe
|
article |
2024/05/10 |
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Rare disorder causes man to see people's faces as 'demonic'
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article |
2024/05/10 |
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In Washington DC and Gaza two very different families are united by one very rare disease
|
article |
2024/05/10 |
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Persistence leads to diagnosis of ultra-rare disease
|
article |
2024/05/10 |
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Pilot program improves well-being of families during advanced care planning
|
article |
2024/05/03 |
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AI Might Spot Rare Diseases in Patients Years Earlier
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article |
2024/05/03 |
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To save lives, the FDA must change how it evaluates rare disease treatments
|
article |
2024/05/03 |
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Imagine living in a 4-foot body that doesn't develop chronic diseases
|
article |
2024/05/03 |
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AI Is Transforming Drug Matching for Cancer, Rare Diseases — Here's How
|
article |
2024/04/27 |
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As more rare disease therapies launch, their prices are rising
|
article |
2024/04/19 |
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Peter Frampton rocks on despite potentially crippling disease: ‘I’m a fighter,’ says the 74-year-old guitar great
|
article |
2024/04/12 |
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Woman with ‘one-of-a-kind’ condition can finally see her own face again after more than 60 hours of surgery
|
article |
2024/04/06 |
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A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access
|
article |
2024/03/21 |
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Gene Therapy for Rare Neurodegenerative Disease Shows Promise in Trial
|
article |
2024/03/21 |
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FDA approves new treatment for rare liver disorder
|
article |
2024/03/14 |
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Big league bartenders a hit at annual fundraiser for rare disease
|
article |
2024/03/14 |
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New way for states to cover pricey gene therapies will start with sickle cell disease
|
article |
2024/03/14 |
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When Should a Pediatrician Suspect a Rare Disease?
|
article |
2024/03/14 |
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My daughter has a rare disease. We shouldn't have had to leave the US to save her life.
|
article |
2024/03/09 |
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Communicating About Rare Diseases
|
article |
2024/03/09 |
|
We Speak Duchenne
|
video |
2024/03/01 |
|
Many parents of kids with rare diseases fight for research — even if it’s too late for their child
|
article |
2024/03/01 |
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Amy Schumer has been diagnosed with Cushing syndrome. What to know about the rare disorder
|
article |
2024/02/25 |
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‘Empowering': Rare Disease Week on Capitol Hill to draw calls for change
|
article |
2024/02/25 |
|
Guest Commentary: Rare diseases affect 30 million Americans. We must answer the challenges to finding cures
|
article |
2024/02/25 |
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What It’s Like to Live With a Rare Liver Disease
|
article |
2024/02/25 |
|
Hailey boy discusses his bout with a rare disease
|
video |
2024/02/17 |
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Opinion: Rare diseases affect 30 million Americans. These are the challenges to find their cure.
|
article |
2024/02/11 |
|
What is Morgellons disease, the mysterious condition Joni Mitchell claims she has?
|
article |
2024/02/04 |
|
What to know about acquired von Willebrand disease (AvWD)
|
article |
2024/01/27 |
|
Ohio mother hopes for a cure to save her son, 8, from rare, fatal disease: ‘Gut-wrenching’
|
article |
2024/01/27 |
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Kernersville woman battling rare disease enrolled in clinical trial
|
article |
2024/01/27 |
|
Rare Disease Patient Photo Contest
|
article |
2024/01/27 |
|
FDA’s Marks Advocates for Flexibility in Rare Disease Gene Therapy Trials
|
article |
2024/01/27 |
|
New CRISPR center brings hope for rare and deadly genetic diseases
|
article |
2024/01/27 |
|
United States Food & Drug Administration (FDA) Grants Mesoblast Rare Pediatric Disease Designation for Revascor® (Rexlemestrocel-L) in Children With Congenital Heart Disease
|
article |
2024/01/20 |
|
Boston Children's Hospital lab trying to develop drug to treat boy's deadly rare disease
|
video |
2024/01/20 |
|
Girl with rare disease 'thriving' after therapy
|
article |
2024/01/17 |
|
FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia
|
article |
2024/01/17 |
|
The Million Dollar Bike Ride for Rare Diseases
|
article |
2024/01/17 |
|
mRNA Technology Emerging for Rare Diseases
|
article |
2024/01/17 |
|
Scientists Discover Rare Mutation That Halves Risk of Parkinson's Disease
|
article |
2024/01/07 |
|
A Doctor And Mother's Plea To FDA To Help Save Children With Rare Disease
|
article |
2024/01/07 |
|
Gene Delivery in Canavan Disease: Innovative Leaps in Technology
|
article |
2023/12/31 |
|
What Is Myelofibrosis?
|
article |
2023/12/31 |
|
Alexander disease: Causes, symptoms, and diagnosis
|
article |
2023/12/23 |
|
Our Impact on Rare Diseases
|
article |
2023/12/17 |
|
FDA approves gene therapy for sickle cell disease: 'One incredible journey of success.'
|
article |
2023/12/12 |
|
Babies with rare disease to survive past first birthday with new drug available on NHS
|
article |
2023/12/03 |
|
Soligenix Receives FDA IND Clearance for Phase 2 Clinical Trial of Dusquetide in the Treatment of Aphthous Ulcers in Behçet's Disease
|
article |
2023/12/03 |
|
Beyond the Diagnosis: Putting a face to children with rare diseases
|
article |
2023/12/03 |
|
7-year-old Bucks County girl with rare disease enjoys holidays for first time thanks to new medication
|
article |
2023/12/03 |
|
Researchers find new therapy that can help treat rare, hereditary diseases
|
article |
2023/11/25 |
|
Pilot launched to support children with rare conditions to access personalised therapies
|
article |
2023/11/25 |
|
Study Highlights Poor Health Outcomes in Older Adults with Hemophilia
|
article |
2023/11/25 |
|
The rare disease films raising greater awareness
|
article |
2023/11/18 |
|
How Common Is Gaucher Disease?
|
article |
2023/11/11 |
|
Rare diseases: Visibility through art
|
article |
2023/11/11 |
|
Overcoming the ongoing challenges for rare disease patients in the UK
|
article |
2023/11/11 |
|
FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder
|
article |
2023/11/11 |
|
Fairport woman with rare disease searches for living liver donor
|
article |
2023/11/04 |
|
The North America Rare Disease Summit focused on the need for centralized data to improve the lives of 30 million people living with rare conditions
|
article |
2023/11/04 |
|
His Rare Disease's Cure Was Sitting on the Pharmacy Shelf
|
article |
2023/11/04 |
|
‘We had no hope’: Patients, advocates testify at U.S. Senate hearing in support of changing FDA rules for rare disease treatments
|
article |
2023/10/27 |
|
Complications You May Experience If You Have Crohn's Disease
|
article |
2023/10/27 |
|
Reps. Kelly, Matsui, Dunn, Thompson introduce legislation to support access to evidence-based care for rare disease patients
|
article |
2023/10/27 |
|
On a journey to diverse, inclusive, and more efficient rare disease trials
|
article |
2023/10/27 |
|
A bright future ahead for rare kidney diseases
|
article |
2023/10/27 |
|
IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases
|
article |
2023/10/27 |
|
National Organization for Rare Disorders Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development
|
article |
2023/10/21 |
|
Former attorney works to bring resources to those with rare genetic disease
|
article |
2023/10/21 |
|
Global Genes and the Rare Disease Diversity Coalition Expand Effort to Accelerate Diagnosis of Rare Disease in Underserved Communities
|
article |
2023/10/21 |
|
Life-changing surgery: Doctor disconnects brain of 6-year-old with rare disease
|
article |
2023/10/15 |
|
National Organization for Rare Disorders Host 2023 Breakthrough Summit
|
article |
2023/10/15 |
|
Baltimore woman defies science, runs marathons with rare autoimmune disease
|
video |
2023/10/15 |
|
Lexi Reed Says She's 'Thankful' for Every Scar: 'I See Battle Wounds'
|
article |
2023/10/15 |
|
Creating an iPS cell resource for rare and intractable diseases
|
article |
2023/10/09 |
|
Novartis says positive interim results on rare kidney disease drug
|
article |
2023/10/09 |
|
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
|
article |
2023/10/01 |
|
Travis Barker says he suffered a facial pain syndrome: What is trigeminal neuralgia?
|
article |
2023/10/01 |
|
VMware: Finding Treatments for Children With Rare Diseases
|
article |
2023/10/01 |
|
American Kidney Fund Launches Educational Awareness Campaigns for Two Rare Kidney Diseases
|
article |
2023/10/01 |
|
Kathleen Folbigg: Misogyny helped jail her, science freed her
|
article |
2023/09/23 |
|
Nearly one in ten Americans will be hit with a “rare” disease
|
article |
2023/09/23 |
|
Canadian Organization for Rare Disorders supports call on United Nations member states to turn universal health coverage into a reality for people living with rare diseases
|
article |
2023/09/23 |
|
Democrat Jennifer Wexton Announces Retirement Following Rare Diagnosis
|
article |
2023/09/19 |
|
How one woman's rare disease experience inspired her career
|
article |
2023/09/16 |
|
'Collectively, we make a big community': 2 Portage residents serve on rare disease council
|
article |
2023/09/16 |
|
Mark Zuckerberg and Dr. Priscilla Chan’s plan to prevent, cure, and manage all diseases by 2100
|
article |
2023/09/16 |
|
Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient
|
article |
2023/09/16 |
|
Small molecule drug shows promise in rare disease
|
article |
2023/09/16 |
|
The Transformative, Alarming Power of Gene Editing
|
article |
2023/09/03 |
|
UC San Diego Launches Gene Therapy Initiative, Targeting Treatments for Rare Diseases
|
article |
2023/09/03 |
|
How whole genome testing saved the life of a baby with a rare disease, and why such tests could help millions more with rare genetic disorders
|
article |
2023/09/03 |
|
Scientists genetically decode rare kidney disease
|
article |
2023/08/26 |
|
A broad genetic test saved one newborn’s life and research suggests it could help millions of others
|
article |
2023/08/26 |
|
What Is Huntington Disease?
|
article |
2023/08/26 |
|
A move to cut drug prices has patients with rare diseases worried
|
article |
2023/08/26 |
|
Rare Diseases
|
podcast |
2023/08/26 |
|
US FDA approves Regeneron's ultra-rare blood disease drug
|
article |
2023/08/19 |
|
4 years later, Ipsen's 'de-risked' rare disease drug Sohonos finally gains FDA approval
|
article |
2023/08/19 |
|
Rare disease patients report great challenges in many areas of life
|
article |
2023/08/19 |
|
3 of 4 children in a Molalla family have rare progressive disorder
|
video |
2023/08/12 |
|
Liver donation brings hope, friendship to 2 Prince William Co. women
|
article |
2023/08/12 |
|
Pennsylvania mother and son both born with rare genetic disease: 'Closer because of this'
|
article |
2023/08/12 |
|
Leprosy Outbreak in Florida: What You Need to Know
|
article |
2023/08/06 |
|
Not So Rare After All: Unveiling the True Prevalence of “Rare” Diseases
|
article |
2023/08/06 |
|
Chinese families being 'destroyed' by burden of facing rare disease ALS
|
article |
2023/08/06 |
|
Opinion: To many treatable diseases go unnoticed. This could change that.
|
article |
2023/08/06 |
|
Teen uses social media to document rare disorder that causes face to waste away
|
video |
2023/07/22 |
|
High-tech clinic in rural Amish community helps children with rare genetic disorders
|
video |
2023/07/22 |
|
Asian countries prioritise rare disease market access amid stark disparities
|
article |
2023/07/22 |
|
Woman With Ultrarare Rare Disease Gives Birth Through IVF in 'World First'
|
article |
2023/07/15 |
|
Solving rare disease mysteries ... and protecting privacy
|
article |
2023/07/15 |
|
Whole Genome Sequencing Boosts Diagnosis of Rare Disease in Infants
|
article |
2023/07/15 |
|
Life saved: AI discovers existing drug works for rare disease
|
article |
2023/07/15 |
|
Early Success: mRNA & CAR T Therapy To Treat Rare Autoimmune Disease Myasthenia Gravis
|
article |
2023/07/09 |
|
Uncommon but Affecting Millions: The Rare Disease Paradox
|
article |
2023/07/04 |
|
FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy
|
article |
2023/06/24 |
|
PTC Therapeutics says interim data for Huntington's disease drug shows promise
|
article |
2023/06/24 |
|
I suffer from the world's most beautiful disease - and also the most expensive to treat
|
article |
2023/06/18 |
|
What is ‘Viking disease’? Deforming hand disorder linked to Neanderthals
|
article |
2023/06/18 |
|
Gunnar Esiason's story highlights importance of clinical trials | Opinion
|
article |
2023/06/11 |
|
SoCal toddler with rare genetic disorder inspires resiliency, research and hope
|
video |
2023/06/11 |
|
Genomics Are a Lifesaver for Patients With Rare Diseases
|
article |
2023/06/11 |
|
What is stiff person syndrome, the condition Celine Dion is battling?
|
article |
2023/05/27 |
|
Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars
|
article |
2023/05/27 |
|
A new research effort takes aim at 8 rare diseases. It could revolutionize many more.
|
article |
2023/05/27 |
|
Widow sheds light on CJD after rare disease takes Michigan man’s life
|
video |
2023/05/21 |
|
Ohio boy, 7, battles rare disease, writes book
|
article |
2023/05/21 |
|
FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa
|
article |
2023/05/21 |
|
Rare Disease: Could Existing Drugs Turn the Tide?
|
article |
2023/05/21 |
|
Mom donates kidney to pediatrician daughter suffering from rare disease
|
video |
2023/05/14 |
|
FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease
|
video |
2023/05/14 |
|
A new, more diverse human genome offers hope for rare genetic diseases
|
article |
2023/05/14 |
|
Michael J. Fox Reveals Private Journey with Parkinson's Disease in Trailer for 'Still' Documentary
|
video |
2023/05/06 |
|
Girl to get life-saving treatment for rare immune disease
|
video |
2023/04/30 |
|
Drug for rare form of Lou Gehrig's disease OK'd by FDA
|
article |
2023/04/30 |
|
Fighting rare diseases: New Hartford boy’s battle with Ohtahara syndrome
|
video |
2023/04/30 |
|
Gene therapy offers hope for rare diseases. But the stories don't always have happy endings.
|
video |
2023/04/30 |
|
Norwich teens manage rare disease phenylketonuria
|
video |
2023/04/30 |
|
How AI Could Make Every Disease A Rare Disease
|
article |
2023/04/22 |
|
Erasing or replacing errors in a patient’s genetic code can treat and cure some genetic diseases
|
article |
2023/04/22 |
|
Family of Naperville girl with Wolf-Hirschhorn Syndrome aims to raise awareness of rare disease
|
video |
2023/04/15 |
|
5,500 people diagnosed with rare genetic disorders in major UK and Ireland study
|
article |
2023/04/15 |
|
6-Year-Old Enjoys Fundraiser for Her Rare Disease: I Wore 'a Unicorn Onesie and Butterfly Wings!'
|
article |
2023/04/08 |
|
Improving Health Equity For Rare Diseases
|
article |
2023/04/08 |
|
How Rare Disease Patients Are Fighting Their Conditions Head-On
|
article |
2023/04/01 |
|
“A Second Chance at Life”: Can Gene Therapies Beat Rare Disease?
|
article |
2023/04/01 |
|
U.S. FDA approves Sanofi's bleeding disorder therapy
|
article |
2023/03/25 |
|
Rare-disease patients battle common issues
|
article |
2023/03/25 |
|
England's NICE signs off on PTC Therapeutics' $3.7M gene therapy for ultra-rare disease
|
article |
2023/03/25 |
|
Genetic causes of three previously unexplained rare diseases identified
|
article |
2023/03/18 |
|
Acadia Awaits Potential Approval of First Rett Syndrome Medicine
|
article |
2023/03/12 |
|
I went from squatting 350 pounds to completely paralyzed at 26 in a matter of weeks. Doctors told me it was a herniated disk, but it was Guillain-Barré.
|
article |
2023/03/12 |
|
Rare disease rap: Patient advocate writes song to raise awareness
|
video |
2023/03/12 |
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I Lost My Daughter To A Rare Disease, But It's What I Did After That Surprises People The Most
|
article |
2023/03/12 |
|
FDA Approves First Therapy for Friedreich's Ataxia (Updated)
|
article |
2023/03/04 |
|
A scientist’s daughter was born with a rare disease, so he began to study it. An anonymous donor just chipped in $25 million
|
article |
2023/03/04 |
|
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.?
|
article |
2023/03/04 |
|
Heartbreak of children lost to rare disease for which there was no cure
|
article |
2023/03/04 |
|
Mental health: Mum with rare disease says support is lacking
|
article |
2023/02/26 |
|
The Reality of Living with a Rare Disease: Emily’s Ongoing Battle
|
article |
2023/02/26 |
|
Explainer: What Is Europe's Rare Disease Moonshot?
|
article |
2023/02/26 |
|
Girl with rare deadly disease receives revolutionary million-dollar gene-therapy drug – but it is too late for her older sister
|
article |
2023/02/20 |
|
Who can afford that'? Patients face costly bills amid FDA's battle over 'orphan drugs'
|
article |
2023/02/20 |
|
A 28-year-old woman thought an old shoulder injury was flaring up. She was actually having mini-strokes due to a rare brain condition.
|
article |
2023/02/12 |
|
A Rare Neurological Disease Involving Cellular Recycling Discovered
|
article |
2023/02/12 |
|
Rare genetic disease may protect Ashkenazi Jews against TB
|
article |
2023/02/12 |
|
2022 was a breakthrough year for understanding rare diseases. 2023 needs to be better
|
article |
2023/02/12 |
|
Bright Ideas for Rare Disease Day 2023
|
article |
2023/02/04 |
|
Huntington's Program Bites the Dust as Novartis Cleans House
|
article |
2023/02/04 |
|
Gene therapy gel heals decades-old wounds in trial for blistering skin disease
|
article |
2023/02/04 |
|
What Causes Parkinson's Disease?
|
article |
2023/01/29 |
|
Gene therapy into brain helps kid with rare disease
|
video |
2023/01/29 |
|
Rare Infectious Diseases: A Tutorial
|
article |
2023/01/29 |
|
Drug repurposing emerges as viable option for rare disease treatment
|
article |
2023/01/29 |
|
Gene therapy gel heals decades-old wounds in trial for blistering skin disease
|
article |
2023/01/29 |
|
Girl with rare disease beats the odds to celebrate 5th birthday
|
article |
2023/01/14 |
|
High school sweethearts tackle rare genetic disease
|
video |
2023/01/14 |
|
Women are finding out their 'rare' health issues aren't actually that uncommon thanks to social media
|
article |
2023/01/14 |
|
Model, 24, with Rare Skin Disease Bares Scars to Raise Awareness: 'I Choose to Show the Body I Was Given'
|
article |
2023/01/07 |
|
Aspen’s story: A 4-year-old living with a rare disease
|
article |
2023/01/07 |
|
Millions have the same ‘bendy body’ disease as my daughter. Why isn’t the medical profession paying more attention?
|
article |
2022/12/30 |
|
Jameela Jamil discusses experience with Ehlers-Danlos syndrome on TikTok
|
article |
2022/12/30 |
|
Oprah Winfrey's Hashimoto's Disease Diagnosis Explained
|
article |
2022/12/30 |
|
Concord woman with rare disease who faced deportation allowed to stay in U.S. permanently
|
article |
2022/12/30 |
|
The Rare Disease Rallying Cry: If Not Me, Who?
|
article |
2022/12/30 |
|
Families Push Research Forward in Rare Diseases
|
article |
2022/12/30 |
|
Rare diseases and space health: optimizing synergies from scientific questions to care
|
article |
2022/12/30 |
|
Man Paralyzed from the Neck Down from Rare Disease Makes Incredible Recovery, Now Back at the Gym
|
article |
2022/12/24 |
|
In a first, children with rare genetic diseases get mitochondrial transplants from their mothers
|
article |
2022/12/24 |
|
The Reasons Why I Share Our Rare Disease Story
|
article |
2022/12/24 |
|
They Created a Drug for Susannah. What About Millions of Other Patients?
|
article |
2022/12/24 |
|
Rare Parents Tackling Rare Diseases
|
article |
2022/12/24 |
|
The Rare World of Rare Diseases
|
article |
2022/12/17 |
|
Sequencing projects will screen 200,000 newborns for disease
|
article |
2022/12/17 |
|
Celine Dion reveals rare neurological disease in emotional video message
|
article |
2022/12/10 |
|
Rare Disease Finding Sheds Light On Head And Neck Cancer
|
article |
2022/12/10 |
|
Missing pathway in lysosome underlies newly discovered human disease
|
article |
2022/12/03 |
|
How AI is finally helping rare diseases gain more than just attention
|
article |
2022/12/03 |
|
What to know about Krabbe disease
|
article |
2022/12/03 |
|
Graves' Disease Explained: Causes Symptoms, And Treatment
|
article |
2022/12/03 |
|
Mental Health and Rare Diseases
|
article |
2022/12/03 |
|
Researchers Track an Ultra-Rare Disease That Turns Muscle to Bone
|
article |
2022/12/03 |
|
Lucy has a rare genetic disorder. Two doctors are leading the desperate hunt for a cure: her parents
|
article |
2022/12/03 |
|
Varun Dhawan To Salman Khan: 5 Bollywood Celebs Who Battled Rare Diseases & Came Out Victorious
|
article |
2022/12/03 |
|
A 27-year-old with a rare disease has died in an experimental gene-editing study. He was the only volunteer.
|
article |
2022/12/03 |
|
With help from Duke doctors, a rare genetic disease is treated in the womb for the first time
|
article |
2022/12/03 |
|
Scientists Finally Discover the Cause of a Rare Brain Disease
|
article |
2022/12/03 |
|
Tiny but mighty' Stamford child with rare joint disease comes home after 5 years in long-term care
|
article |
2022/09/24 |
|
For Some ALS Patients, A New Drug Shows Potential To Slow And Even Reverse Disease Progression
|
article |
2022/09/24 |
|
New uses for old drugs? Every Cure offers hope for people with rare diseases
|
article |
2022/09/24 |
|
FDA, NIH Launch Public-private Partnership For Rare Neurodegenerative Diseases
|
article |
2022/09/17 |
|
Scientists discover novel mechanism that causes rare brain disease
|
article |
2022/09/17 |
|
U.S. FDA approves bluebird bio's gene therapy for a rare neurological disorder
|
article |
2022/09/17 |
|
New Parkinson’s test developed thanks to woman who could smell the disease
|
article |
2022/09/10 |
|
What to Know About Capillary Leak Syndrome
|
article |
2022/09/10 |
|
Sanofi, after long research journey, wins FDA approval for rare disease drug
|
article |
2022/09/02 |
|
Rare Disease Report Podcast: CDKL5 Deficiency Disorder
|
podcast |
2022/09/02 |
|
The Most Expensive Drug in the US is a Rare-Disease Game Changer
|
article |
2022/08/28 |
|
San Diego woman with rare disease receives first breakthrough treatment at UCSD
|
video |
2022/08/28 |
|
What is Rett syndrome? Richard Engel mourns death of 6-year-old son Henry
|
article |
2022/08/21 |
|
bluebird bio Announces FDA Approval of ZYNTEGLO®, the First Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions
|
article |
2022/08/21 |
|
Drug Development for Rare Diseases is Littered with Regulatory Roadblocks
|
article |
2022/08/21 |
|
‘Abnormal’ Protein Could Be Common Link Between All Forms of Motor Neuron Disease
|
article |
2022/08/14 |
|
Cure Rare Disease Scores IND for First-in-Human CRISPR Therapeutic
|
article |
2022/08/14 |
|
Couple speaks out on decision to get abortion after fetus diagnosed with rare genetic conditions
|
video |
2022/08/10 |
|
What You Need to Know About This Rare Chronic Autoimmune Disease
|
article |
2022/08/07 |
|
Charlotte family launches foundation to raise awareness of rare disease
|
video |
2022/08/07 |
|
Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne Muscular Dystrophy
|
article |
2022/08/07 |
|
Designing a tool for better diagnosis of rare and genetic diseases
|
article |
2022/08/07 |
|
Gaining insights into spastic paraplegia
|
article |
2022/08/07 |
|
Poor health-related quality of life can lead to rare diseases
|
article |
2022/08/07 |
|
Millions of people have rare diseases, including my son. Here’s how Congress can help
|
article |
2022/07/30 |
|
Medical Informatics and Rare Disease: a bridge between two worlds
|
article |
2022/07/30 |
|
The Integration of Gene Therapy for Rare Disease
|
article |
2022/07/30 |
|
Parents are becoming drug developers to find a cure for their children’s rare diseases
|
article |
2022/07/24 |
|
People with Rare Diseases Need Better Healthcare
|
article |
2022/07/24 |
|
There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be
|
article |
2022/07/24 |
|
CONSTANT AGONY Page 3 Girl Michelle Marsh reveals desperate battle to save her daughter’s life from rare disease
|
article |
2022/07/24 |
|
Study highlights the multibillion dollar burden of rare disease
|
article |
2022/07/24 |
|
Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years
|
article |
2022/07/24 |
|
Brad Pitt believes he has rare 'face blindness' disorder—what is it?
|
article |
2022/07/16 |
|
Rare Diseases in India: ‘Orphan’ No More?
|
article |
2022/07/16 |
|
Finding Comfort in the Rare Disease Community
|
article |
2022/07/09 |
|
Drilling for rare disease therapeutics
|
article |
2022/07/09 |
|
Rare diseases: "Recognition of a disability is still complicated in Europe."
|
article |
2022/07/09 |
|
Lab-grown 'mini-kidneys' unlock secrets of a rare disease
|
article |
2022/07/09 |
|
What's Up Doc? Rare skin disorder causes blistering, calls for genetic counseling
|
article |
2022/07/01 |
|
Report finds thousands of uncounted rare diseases in challenge to current estimates
|
article |
2022/06/19 |
|
What Is Ramsay Hunt Syndrome? What to Know About Justin Bieber's Rare Disease
|
article |
2022/06/11 |
|
People with rare diseases have poor health-related quality of life, study finds
|
article |
2022/06/11 |
|
What is Cushing's disease? Experts warn of 'rare but serious' condition
|
article |
2022/06/11 |
|
Is your child at higher risk of rare disease? Why more couples are going for genetic tests
|
article |
2022/06/04 |
|
The Rare Disease Report Podcast: Myelodysplastic Syndromes & Acute Myeloid Leukemia
|
article |
2022/06/04 |
|
Castleman Disease: Types, Symptoms, and Treatment
|
article |
2022/05/22 |
|
Musician with rare disease overcomes health scare
|
article |
2022/05/22 |
|
Francis Collins Urges Gene Therapy Community to Scale Efforts to Tackle Rare Diseases
|
article |
2022/05/22 |
|
CDER Launches New Accelerating Rare disease Cures (ARC) Program
|
article |
2022/05/15 |
|
Lawmakers discuss push for rare disease prevention, support
|
article |
2022/05/15 |
|
Takeda scraps dream of getting rare disease drug to market in latest setback to Wave 1 pipeline
|
article |
2022/05/15 |
|
Why Pompe Disease Can Be Mistaken for Other Genetic Diseases
|
article |
2022/05/08 |
|
Cilia-free stem cells offer new path to study rare diseases
|
article |
2022/05/08 |
|
Undiagnosed Patient Organizations Begin Leveraging RARE-X
|
article |
2022/05/01 |
|
Editas Medicine Gets FDA Rare Pediatric Disease Designation for EDIT-301
|
article |
2022/05/01 |
|
Organizations Rally to Help Ukrainian Rare Disease Patients
|
article |
2022/05/01 |
|
Cautions issued over prenatal genetic tests in IVF and for rare diseases
|
article |
2022/05/01 |
|
When it comes to the rarest of diseases, the diagnosis isn't the answer – it's just the starting point
|
article |
2022/04/23 |
|
A dangerous delay: Why it can take years to diagnose life-threatening rare diseases
|
article |
2022/04/23 |
|
Drug development for ultra-rare diseases: What happens when N=1?
|
article |
2022/04/23 |
|
AAAS Webinar-- Reimagining rare disease detection: Who needs to be at the table?
|
video |
2022/04/16 |
|
American Kidney Fund Applauds Introduction of New Era for Preventing End-Stage Kidney Disease Act of 2022
|
article |
2022/04/16 |
|
Myotonic Dystrophy Is a Rare, Genetic Disease—And Its Link to Ventricular Tachycardia Is Even Rarer
|
article |
2022/04/16 |
|
People with alopecia share their stories: ‘Hair does not define any of us’
|
video |
2022/04/14 |
|
My baby was born with a rare congenital disease, and I didn't realize until he was 2 months old. Googling his symptoms helped me advocate for him.
|
article |
2022/04/10 |
|
Gene-therapy gel shows promise for blistering skin disease in clinical trial
|
article |
2022/04/07 |
|
Woman with rare illness that's seen her dislocate 'hundreds' of bones can't afford to stay at university
|
article |
2022/04/02 |
|
Kids with rare autoimmune disease show these symptoms before blood clots
|
article |
2022/03/27 |
|
Ascentage Pharma's MDM2-p53 Inhibitor Alrizomadlin (APG-115) Granted Rare Pediatric Disease Designation By The US FDA For The Treatment Of Neuroblastoma
|
article |
2022/03/27 |
|
Doctor Who Devised a Treatment for Her Rare, Painful Disease Started Clinic to Help Others
|
article |
2022/03/27 |
|
Increased Education Crucial to Improving Rare Disease Care, Survey Finds
|
article |
2022/03/27 |
|
For people living with rare diseases, waiting on the STAT Act can be frustrating
|
article |
2022/03/20 |
|
I've had a blood transfusion every 3 weeks since I was 2 months old due to a rare disease that can kill people in their 30s
|
article |
2022/03/20 |
|
Climbing Mount Everest? A courageous dad fundraises to find cure for a muscle disease in hopes of saving son’s life.
|
article |
2022/03/13 |
|
Science Brings Shortcut to Spotting 50 Rare Genetic Diseases
|
article |
2022/03/13 |
|
Science Brings Shortcut to Spotting 50 Rare Genetic Diseases
|
article |
2022/03/13 |
|
Rare Disease Day: Parents share arduous experience, hopeful future in raising children with rare diseases
|
article |
2022/03/05 |
|
My Daughter's Rare Disease Was A Mystery For Years. Here's How We Finally Got A Diagnosis.
|
article |
2022/03/05 |
|
It’s a rare disease affecting 200,000 women, no cure, and no one is talking about it until now
|
video |
2022/03/05 |
|
Cell research on rare disease finds new link to inflammation
|
article |
2022/03/05 |
|
Rare Disease Symposium connects families, doctors and scientist looking for answers
|
article |
2022/03/05 |
|
Report: Economic burden of rare diseases is 10 times higher than mass market diseases
|
article |
2022/03/05 |
|
New FDA program could boost drug development for rare diseases
|
article |
2022/02/22 |
|
Early Trial Offers Hope Treating Rare 'Brittle Bone' Disease
|
article |
2022/02/22 |
|
First Gene Therapy For Tay-Sachs Disease Successfully Given To Two Children
|
article |
2022/02/22 |
|
Record-breaking rapid DNA sequencing promises timely diagnosis for thousands of rare diseases
|
article |
2022/02/06 |
|
Parents Lose Two Babies Weeks Apart as Child Dies of Rare Genetic Disease
|
article |
2022/02/06 |
|
There's no cure for rare types of cystic fibrosis, but researchers are making significant advances
|
article |
2022/02/06 |
|
Scientists identify new features of lymphangioleiomyomatosis, a rare lung disease
|
article |
2022/02/06 |
|
We in the Rare Disease Community Can Learn From Each Other
|
article |
2022/02/06 |
|
My baby was born with disease so rare it doesn’t have a name – I’m desperate to find cure before he dies
|
article |
2022/01/29 |
|
Analysis reveals rare respiratory disease PCD is more common than previously thought
|
article |
2022/01/29 |
|
Parents Refuse To Take No For An Answer When Son Is Diagnosed With Incurable Disease
|
article |
2022/01/22 |
|
Herpes virus "likely" main cause of Multiple Sclerosis study finds
|
article |
2022/01/15 |
|
Hutchinson-Gilford Progeria Syndrome: What to Know About the Rare and Fatal Genetic Disorder
|
article |
2022/01/15 |
|
What to Know About Scleroderma, the Autoimmune Disease Bob Saget Fought for Before His Death
|
article |
2022/01/15 |
|
Children With Rare Fatal Disease Trial World-First Treatment To Save Their Sight
|
article |
2022/01/15 |
|
The Colorado Parents Fighting the World’s Rarest Diseases
|
article |
2022/01/01 |
|
Diagnostic odyssey: The lonely road walked by thousands of Coloradans with disorders so rare even their doctors hadn't heard of them
|
article |
2022/01/01 |
|
First-ever United Nations Resolution to Increase Visibility for the 300 Million Persons Living with a Rare Disease
|
article |
2021/12/19 |
|
Treatment in Texas: For families of kids with rare diseases, it’s a full-time job to advocate for, raise millions for research
|
video |
2021/12/12 |
|
Rare gene in Amish people helps protect their hearts
|
article |
2021/12/05 |
|
24-Year-Old Researches Treatment for Her Own Crippling Disease: 'I'm in a Race Against Time'
|
article |
2021/12/05 |
|
Gene-Based Therapy for Rare Skin Disease Succeeds in Phase III
|
article |
2021/11/30 |
|
New treatment for a rare genetic disease to be made available on NHS
|
article |
2021/11/20 |
|
General Hospital' star Bergen Williams dead at 62 after battling rare Wilson's disease
|
article |
2021/11/20 |
|
Whole genome sequencing improves diagnosis of rare diseases and shortens diagnostic journeys for patients
|
article |
2021/11/14 |
|
Hundreds of patients in gene study given rare disease diagnosis
|
article |
2021/11/14 |
|
Rare Genetic Mutation in Utah Family Traced Across Continents And Over Centuries
|
article |
2021/11/14 |
|
FDA Approves Treatment for Rare Blood Disease
|
article |
2021/11/14 |
|
Australia's first snapshot of families at risk of blindness from rare genetic eye disease
|
article |
2021/11/06 |
|
CHOC and UCI Health Join New NORD Rare Disease Centers of Excellence Network, Committed to Improving Access and Care for Rare Disease Patients
|
article |
2021/11/06 |
|
National Organization for Rare Disorders recognizes OU Health as Rare Disease Center of Excellence
|
article |
2021/11/06 |
|
https://www.oudaily.com/news/national-organization-for-rare-disorders-recognizes-ou-health-as-rare-disease-center-of-excellence/article_f7e54766-3db2-11ec-a63d-3f53633a7cf1.html
|
article |
2021/11/06 |
|
FDA, NIH Announce Bespoke Gene Therapy Consortium to Focus on Rare Diseases
|
article |
2021/10/30 |
|
NIH study suggests people with rare diseases face significantly higher health care costs
|
article |
2021/10/24 |
|
FDA awards 11 clinical trial grants for rare diseases, many in children
|
article |
2021/10/16 |
|
Krystal Biotech and GeneDx Announce Collaboration to Provide No-charge Genetic Testing for Patients with Suspected Dystrophic Epidermolysis Bullosa (DEB)
|
article |
2021/10/16 |
|
New stem cell source offers hope to patients with rare liver disease
|
article |
2021/10/16 |
|
What to know about paroxysmal nocturnal hemoglobinuria (PNH)
|
article |
2021/10/16 |
|
Rare Disease Diversity Coalition Awards $600K to Combat Disparities
|
article |
2021/10/16 |
|
Is technology the key to accelerating rare disease clinical trials?
|
article |
2021/10/16 |
|
AI Rapidly Diagnoses Rare Disorders in Critically Ill Children
|
article |
2021/10/16 |
|
ChemoCentryx's drug gets U.S. FDA nod for treating rare autoimmune disease
|
article |
2021/10/10 |
|
Mirum Snags First Drug Approval for Rare, Genetic Liver Disorder
|
article |
2021/10/03 |
|
Yeast Models Provide New Insights into Neurodegenerative Diseases
|
article |
2021/10/03 |
|
New Data-sharing Program Aims to Speed Innovation in Rare Diseases
|
article |
2021/10/03 |
|
Actors Who Have Rare Diseases And Medical Conditions
|
article |
2021/10/03 |
|
Rare Diseases 2021: running rare disease trials post-Covid
|
article |
2021/09/25 |
|
Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness
|
article |
2021/09/25 |
|
Rare Disease Spotlight: Sarcoidosis
|
article |
2021/09/25 |
|
Moderna to develop mRNA therapeutic for ultra-rare disease
|
article |
2021/09/25 |
|
8-Year-Old Girl Is Left Paralyzed After COVID Likely Triggered Rare Disease
|
article |
2021/09/19 |
|
The Champion for Rare Disease Cures
|
article |
2021/09/19 |
|
California legislature poised to establish Rare Disease Advisory Council
|
article |
2021/09/19 |
|
MMS support program to help ultra-rare disease research
|
article |
2021/09/19 |
|
Moderna donates ultra-rare disease therapy to nonprofit founded by late Takeda R&D chief
|
article |
2021/09/11 |
|
New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many
|
article |
2021/09/11 |
|
British father begins 1,200- mile barefoot walk in Maine to raise awareness for daughter's rare disorder
|
video |
2021/08/29 |
|
A father on the legacy of his son's ultra-rare disease
|
podcast |
2021/08/29 |
|
AstraZeneca drug for rare disease shows promise in flushing copper build-up
|
article |
2021/08/29 |
|
BioMarin stands tall with first approved drug for rare disease that causes dwarfism
|
article |
2021/08/29 |
|
‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases
|
article |
2021/08/29 |
|
Parents create nonprofit to find cure for toddler's rare disease
|
video |
2021/08/22 |
|
8-year-old Camryn Kellam Beating Back Blood Disease, Rare Brain Syndrome
|
video |
2021/08/22 |
|
Woman hits the road to support Rare Disease Cause
|
video |
2021/08/22 |
|
This Gainsville mom wants to raise awareness of rare disease with upcoming walk at Wilshire Trail
|
article |
2021/08/17 |
|
For two UB scientists, love means studying daughter’s rare disease
|
article |
2021/08/08 |
|
With a nudge from AI, ketamine emerges as a potential rare disease treatment
|
article |
2021/08/08 |
|
Rare disorder offers roadmap for understanding roots of inflammatory disease
|
article |
2021/07/31 |
|
‘I Just Want My Kid To Have A Good Life’: Colorado Boy 1 In 40 Worldwide Diagnosed With Rare Disease
|
video |
2021/07/31 |
|
Cecilia woman testifies to Congress about rare disease
|
article |
2021/07/31 |
|
Healx: AI-driven drug repurposing for rare disease
|
article |
2021/07/24 |
|
Is Artificial Intelligence The Best Solution For Diagnosing Rare Diseases?
|
article |
2021/07/18 |
|
Early Study Shows Promise of CRISPR Injection to Treat Rare Disease
|
article |
2021/07/18 |
|
NIH-funded study finds gene therapy may restore missing enzyme in rare disease
|
article |
2021/07/18 |
|
Photographs reveal people behind rare illnesses
|
article |
2021/07/11 |
|
Dying patients with rare diseases struggle to get experimental therapies
|
video |
2021/07/11 |
|
Community rallies around 6-year-old with rare disease. His family needs your help.
|
article |
2021/07/11 |
|
After Timesia Hart Was Diagnosed With a Rare Disease, She Started a Bike Race to Inspire Others
|
article |
2021/07/07 |
|
Legislation to Help Diagnose Children with Rare Diseases Introduced by Senators Collins, Kelly, and Menendez
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article |
2021/06/27 |
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Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines
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article |
2021/06/20 |
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The Loneliness I Feel as the Parent of a Child With a Rare Disease
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article |
2021/06/20 |
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Columbus mom lost one daughter to a rare disease. A new drug is helping another
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video |
2021/06/12 |
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KC family worries cost could keep drug from helping son with rare disorder
|
video |
2021/06/12 |
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Raleigh biopharma company hopes to have a new treatment for celiac disease
|
article |
2021/06/05 |
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Unprecedented data sharing driving new rare disease diagnoses in Europe
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article |
2021/06/05 |
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FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder
|
article |
2021/06/05 |
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The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price
|
article |
2021/06/05 |
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Newer methods may boost gene therapy's use for more diseases
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article |
2021/06/05 |
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Child fights for her life by taking the 'most expensive drug in the world'
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video |
2021/05/21 |
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‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access
|
article |
2021/05/21 |
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Las Vegas woman writes children's book to help people with rare disease
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video |
2021/05/21 |
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Treating Polycythemia Vera
|
article |
2021/05/16 |
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Rare diseases the next target for mRNA therapies
|
article |
2021/05/16 |
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Why I Hate My Kid's Birthday
|
article |
2021/05/16 |
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Moms search for lifesaving bone marrow donors for daughters with rare diseases
|
article |
2021/05/16 |
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Rare Disease Companies Unite to Advocate for Life-Changing Therapies
|
article |
2021/05/16 |
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Cystic Fibrosis: One Patient’s Story, Current Treatments and Exciting New Therapies on the Horizon
|
article |
2021/05/16 |
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Gap in care, research and funding for rare blood disorder disproportionately affecting Black Americans
|
video |
2021/05/09 |
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CENTOGENE Discovers Six New Rare Diseases by Leveraging the Strength of Its Bio/Databank
|
article |
2021/05/09 |
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Virtual gala to raise funds, awareness for rare neurodevelopmental disorder
|
article |
2021/05/02 |
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Chiesi Global Rare Diseases Announces FDA Approval of FERRIPROX® (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease
|
article |
2021/05/02 |
|
Woman with rare muscular disease sings to exercise her lungs. Her songs inspire others to chase their dreams
|
video |
2021/05/02 |
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CDISC and NORD Partner to Develop Data Standards for Rare Diseases
|
article |
2021/04/24 |
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A new treatment for rare muscular disease
|
article |
2021/04/17 |
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Detecting rare disease: Revealing the methods, motivations, and implications
|
article |
2021/04/17 |
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EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
|
article |
2021/04/17 |
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Opinion: Genome sequencing can lead to life-changing care for infants. California should make it more available
|
article |
2021/04/10 |
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Keenan: Reseachers are 'in this together' to solve rare diseases
|
article |
2021/04/10 |
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Ducks’ Brady Breeze ‘repping for rare diseases’ at pro day
|
video |
2021/04/03 |
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Software engineer bets on technology to help speed rare disease treatments
|
article |
2021/04/03 |
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Scientists discover new genetic disease that delays brain development in children
|
article |
2021/04/03 |
|
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
|
article |
2021/04/03 |
|
The poor diagnosis of rare diseases: Overcoming deficits in information, awareness, and understanding
|
article |
2021/03/27 |
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Report: Orphan Drug Act Boosted Rare Disease Therapies, But Access is Uneven
|
article |
2021/03/27 |
|
STAT Act Offers Hope to People Living with Rare Diseases
|
article |
2021/03/21 |
|
COVID's all-hands-on-deck approach should be standard for rare diseases
|
article |
2021/03/21 |
|
How Covid-19 is changing rare diseases research
|
article |
2021/03/21 |
|
More than a quarter of rare disease trials are culled due to low patient rates: report
|
article |
2021/03/13 |
|
How Covid-19 is changing rare diseases research
|
article |
2021/03/13 |
|
Raising awareness for rare diseases matters every day
|
article |
2021/03/07 |
|
‘You can carry on or give up’: Families living with rare diseases
|
article |
2021/03/07 |
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Push to spur more drugs for deadly rare diseases
|
article |
2021/03/07 |
|
Rare diseases in the Bronze Age
|
article |
2021/03/06 |
|
Most dollars spent on top-selling orphan drugs don’t go to treat people with rare diseases
|
article |
2021/03/06 |
|
Stars who suffer from rare diseases
|
article |
2021/02/28 |
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7,000 challenges: The basis and burden of rare diseases
|
article |
2021/02/28 |
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FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A
|
article |
2021/02/28 |
|
CDER’s Progress in Rare Diseases
|
article |
2021/02/28 |
|
Devastated mum loses all three kids to rare disease found in just 110 people in UK
|
article |
2021/02/21 |
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Guest column: Innovative testing detects rare diseases in critically ill infants
|
article |
2021/02/21 |
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Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases
|
article |
2021/02/21 |
|
Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results
|
article |
2021/02/13 |
|
Early study points to potential therapeutic avenue for a pair of rare pediatric diseases
|
article |
2021/02/13 |
|
Rare congenital malformation caused by epigenetic mechanism in previously mysterious genome sequences
|
article |
2021/02/13 |
|
How the human genome transformed study of rare diseases
|
article |
2021/02/13 |
|
Islanders embrace families whose children have rare diseases
|
article |
2021/02/13 |
|
Pandemic Won’t Stop Rare Disease Day on Feb. 28
|
article |
2021/02/13 |
|
Global Liver Institute Announces First Annual Rare Liver Diseases Month
|
article |
2021/02/08 |
|
Canadians Invited To Share Their Views On A National Strategy For High-Cost Drugs For Rare Diseases
|
article |
2021/01/31 |
|
A patient-powered registry boosts the study of rare Castleman disease
|
article |
2021/01/31 |
|
Partners in crime: Genetic collaborator may influence severity of the rare disease, NGLY1
|
article |
2021/01/31 |
|
NORD and Partners Host FDA, CDC Leaders in Webinar Addressing Rare Disease Community on COVID-19 Vaccines
|
article |
2021/01/31 |
|
RECORDATI RARE DISEASES: CARBAGLU® (Carglumic Acid) Tablets 200mg Receives U.S. FDA Approval for a New Indication to Treat Acute Hyperammonemia Associated with Propionic Acidemia and Methylmalonic Acidemia
|
article |
2021/01/31 |
|
Researchers use patients’ cells to test gene therapy for rare eye disease
|
article |
2021/01/31 |
|
Rare Artist 2020 award recipients
|
webpage |
2021/01/31 |
|
FDA Aims to Ease Single-Patient Trials for Rare Disease Drugs (1)
|
article |
2021/01/31 |
|
Study Pins Down Number of Americans with Most Common Form of Lupus
|
article |
2021/01/31 |
|
ERN-LUNG Project Helping People With Rare Lung Diseases
|
article |
2021/01/31 |
|
M6P Therapeutics Receives Six Rare Pediatric Disease Designations from the U.S. FDA for Company’s Deep Pipeline of Programs for Lysosomal Storage Disorders
|
article |
2021/01/31 |
|
Taysha Gene Therapies Receives Rare Pediatric Disease And Orphan Drug Designations For TSHA-105 For The Treatment Of Epilepsy Caused By SLC13A5 Deficiency
|
article |
2021/01/24 |
|
COVID Vaccine for Patient With Rare Disease? Best Guess Is Yes
|
article |
2021/01/24 |
|
New taxonomy of non-skeletal rare disorders with impact on bone
|
article |
2021/01/16 |
|
Using DNA sequencing data with electronic health records to find rare variants behind inherited diseases
|
article |
2021/01/16 |
|
The UK Rare Diseases Framework
|
article |
2021/01/16 |
|
Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for Pegunigalsidase Alfa for Proposed Treatment of Fabry Disease
|
video |
2021/01/16 |
|
I don't know of anyone else': Man with rare disease pens book to help others
|
video |
2021/01/10 |
|
Girl with Uncombable Hair Syndrome is finally able to have her hair brushed after nine years
|
article |
2021/01/10 |
|
Researchers create an animal model for studying GPI anchor deficiencies
|
article |
2021/01/10 |
|
Could CRISPR Be the Key for Rapidly Aging Children?
|
article |
2021/01/10 |
|
Top 5 Most-Read Rare Disease Stories of 2020
|
article |
2021/01/03 |
|
How an ultra-rare disease is bringing this N.S. family closer together
|
article |
2021/01/03 |
|
11 rare skin diseases
|
article |
2020/12/27 |
|
How to Rethink Population Health Management for Rare Disease Care
|
article |
2020/12/27 |
|
Community Hero: Grant Bonebrake champions those with rare diseases and wins national award
|
article |
2020/12/21 |
|
Emedgene collaborates with Illumina to scale the interpretation of genomic data for rare diseases
|
article |
2020/12/21 |
|
No Deal Brexit could endanger lives of four million Brits with rare diseases
|
article |
2020/12/13 |
|
£1m step closer to understanding genetic diseases
|
article |
2020/12/13 |
|
Treatments for rare diseases at risk, due to the decline of plasma donations
|
article |
2020/12/13 |
|
Local nonprofit helping patients with ultra-rare diseases
|
article |
2020/12/13 |
|
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
|
article |
2020/12/13 |
|
View from inside: Rare diseases in the times of COVID19
|
article |
2020/12/13 |
|
The rare condition slowly paralysing my arms and legs
|
article |
2020/12/06 |
|
Fulcrum Therapeutics Presents Updated Data On Sickle Cell Disease Program At The 62nd American Society Of Hematology (ASH) Annual Meeting And Exposition
|
article |
2020/12/06 |
|
People with rare autoimmune diseases at increased risk of dying during COVID-19 pandemic
|
article |
2020/12/06 |
|
Aeglea BioTherapeutics Receives FDA Rare Pediatric Disease Designation For ACN00177 For The Treatment Of Homocystinuria
|
article |
2020/12/06 |
|
Genethon: green light from the ANSM to start a gene therapy trial for Duchenne Dystrophy
|
article |
2020/12/06 |
|
How the rare disease community has developed fertile ground for progress
|
article |
2020/11/29 |
|
FDA Approves First Treatment For Hutchinson-Gilford Progeria Syndrome And Some Progeroid Laminopathies
|
article |
2020/11/22 |
|
Eddie Vedder Leads Star-Studded Event To Raise Awareness And Funds For Rare Disease
|
article |
2020/11/15 |
|
Cystic Fibrosis
|
article |
2020/11/15 |
|
How people in China with rare diseases afford outrageously expensive medicines?
|
article |
2020/11/09 |
|
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
|
article |
2020/11/09 |
|
Diagnoses of rare diseases enhanced through the teamwork of national network
|
article |
2020/11/01 |
|
Identifying and recruiting patients into rare disease clinical trials: A multi-pronged and customized approach
|
article |
2020/11/01 |
|
Retrophin buys up Orphan Technologies, spending $90M on rare disease drug
|
article |
2020/10/24 |
|
Vaccines and rare diseases: using one to help the other
|
article |
2020/10/24 |
|
Study provides new hope for children suffering from rare muscle diseases
|
article |
2020/10/18 |
|
Researchers lead national efforts around childhood blood disorders
|
article |
2020/10/18 |
|
People With Rare Diseases Need Better Social Support
|
article |
2020/10/18 |
|
BridgeBio Pharma And Affiliate Origin Biosciences Announces FDA Acceptance Of Its New Drug Application For Fosdenopterin For The Treatment Of MoCD Type A
|
article |
2020/10/11 |
|
Providing new hope for children suffering from rare muscle diseases
|
article |
2020/10/11 |
|
I ran from my lethal diagnosis for years. Let's make this rare disease a household word.
|
article |
2020/10/11 |
|
Woman with rare disease delivers healthy baby
|
article |
2020/10/11 |
|
Flying the flag for research in Aarskog syndrome
|
article |
2020/10/11 |
|
Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD
|
article |
2020/10/11 |
|
Richard Engel Celebrates Son's 5th Birthday, Shares Message to All Kids with Rett Condition
|
article |
2020/10/03 |
|
New osteosarcoma treatment receives rare pediatric disease designation from the FDA
|
article |
2020/10/03 |
|
Lorain County family devoted to patient advocacy with rare disease foundation
|
article |
2020/10/03 |
|
Reauthorize the Creating Hope Act to help kids with rare diseases
|
article |
2020/09/27 |
|
FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis
|
article |
2020/09/27 |
|
Skeletal Rare Diseases Academy launched by IOF
|
article |
2020/09/19 |
|
Understanding genetics of common, rare disorders advance treatment in pediatric neurology
|
article |
2020/09/19 |
|
Metopirone Safely Normalized, or Significantly Lowered, Urine Cortisol Levels in Phase 3/4 Trial
|
article |
2020/09/19 |
|
Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease
|
article |
2020/09/19 |
|
The needs of disabled children are being overlooked in the wake of covid-19
|
article |
2020/09/19 |
|
Newborn Screening Practices and Alpha-Thalassemia Detection — United States, 2016
|
article |
2020/09/12 |
|
Rallying Toward a Better Future for Pompe Disease
|
article |
2020/09/12 |
|
Same But Different Photo Contest Celebrates People With Rare Diseases
|
article |
2020/09/12 |
|
Customized gene therapies successfully target rare eye diseases
|
article |
2020/09/12 |
|
A race against time: Young boy and his family cope with rare disease
|
article |
2020/09/12 |
|
Getting a Treatment Is Not the End of the Road in Rare Diseases
|
article |
2020/09/06 |
|
Using telehealth to revolutionize the speed of making rare disease diagnoses
|
article |
2020/08/30 |
|
Rare Diseases: My teeth fall off easily and my bones are so hard walking breaks them
|
article |
2020/08/30 |
|
FDA Approves Treatment for Rare Disease Affecting Optic Nerves, Spinal Cord
|
article |
2020/08/24 |
|
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
|
article |
2020/08/24 |
|
Kubota Vision Receives Orphan Products Clinical Trials Grants to Emixustat for Stargardt Disease
|
article |
2020/08/24 |
|
FDA Grants Rare Pediatric Disease Designation to AmideBio’s Treatment for Congenital Hyperinsulinism
|
article |
2020/08/24 |
|
Peer support groups help mum of child with a rare liver disease find answers and support
|
article |
2020/08/24 |
|
Chronic Lyme disease is a rare, but serious condition — here's how to know if you have it
|
article |
2020/08/24 |
|
Barefoot major finishes 700 mile walk for daughter
|
article |
2020/08/15 |
|
Enrollment in the U.S. Phase 2 Clinical Trial in Cystic Fibrosis Has Resumed After Being Paused in Response to the COVID-19 Pandemic
|
article |
2020/08/15 |
|
New Streaming Channel Showcases Rare Disease Films
|
article |
2020/08/15 |
|
10 Rare Autoimmune Diseases That Don’t Get Enough Attention
|
article |
2020/08/15 |
|
How Can I Best Prepare for an ABA Therapist in My Home?
|
article |
2020/08/11 |
|
EU support for Italian biotech's red blood cell technology to treat rare diseases
|
article |
2020/08/08 |
|
Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest
|
article |
2020/08/08 |
|
FDA Approves Oral Treatment for Spinal Muscular Atrophy
|
article |
2020/08/07 |
|
FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease
|
article |
2020/08/04 |
|
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived
|
article |
2020/08/02 |
|
Voices from the front line in rare disease
|
article |
2020/08/02 |
|
Potential treatment for rare degenerative disease discovered
|
article |
2020/08/02 |
|
Rare Diseases in Asia-Pacific
|
article |
2020/08/02 |
|
Researchers develop new therapeutic approach to treat CLN3 Batten disease
|
article |
2020/08/02 |
|
FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL
|
article |
2020/07/29 |
|
My Constant Insomnia Turned Out to Be a Symptom of a Rare Disorder Called Hypophosphatasia
|
article |
2020/07/29 |
|
How coronavirus affects young adults with chronic illnesses and rare diseases
|
article |
2020/07/26 |
|
Rare disease drove two women on a mission to change FDA drug review process
|
article |
2020/07/19 |
|
CZI awards $1.3 million to support the global rare disease community
|
article |
2020/07/19 |
|
'It Will Consume Your Life': 4 Families Take On Rare Diseases
|
article |
2020/07/11 |
|
Army major walking barefoot from Cornwall to Edinburgh to fund treatment for daughter with rare disease
|
article |
2020/07/11 |
|
FDA Approves New Therapy for Myelodysplastic Syndromes (MDS) That Can Be Taken at Home
|
article |
2020/07/07 |
|
Two Patient Deaths Halt Audentes’ Gene Therapy Trial
|
article |
2020/07/02 |
|
What My Family and Friends Want You to Know About Mal de Debarquement Syndrome
|
article |
2020/07/01 |
|
Lewisville family helps to raise awareness for rare disease
|
article |
2020/07/01 |
|
A Helpful Online Safety Guide for People With Autism Spectrum Disorders
|
article |
2020/07/01 |
|
A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving
|
podcast |
2020/06/27 |
|
FDA Approves New Therapy for Dravet Syndrome
|
article |
2020/06/25 |
|
I want to cure my son of his rare genetic disease. Is that wrong?
|
article |
2020/06/21 |
|
Meet the family working with scientists to fight rare diseases with CRISPR therapy
|
video |
2020/06/21 |
|
My friend chose an assisted death in Switzerland. Her dying wish was to tell you why
|
article |
2020/06/21 |
|
FDA Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease
|
article |
2020/06/16 |
|
Coalition Will Address Racial Disparities in Rare Disease Communities
|
article |
2020/06/14 |
|
Sobi and Sanofi Donate up to 500 Million Additional IUs of Clotting Factor to WFH Humanitarian Aid Program
|
article |
2020/06/14 |
|
Recordati Rare Diseases Inc. Announces Availability of ISTURISA® (osilodrostat) in the United States
|
article |
2020/06/14 |
|
FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord
|
article |
2020/06/14 |
|
Rare Disease Therapy Development and Access Remain Top FDA Priorities During COVID-19
|
article |
2020/06/11 |
|
FDA Approves Orphan Drug Pemigatinib for Rare Bile Duct Cancer Cholangiocarcinoma
|
article |
2020/06/10 |
|
Study reveals biochemical alterations in patients with Lesch-Nyhan disease
|
article |
2020/06/10 |
|
Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports
|
article |
2020/06/10 |
|
Drug offers hope for patients with primary hyperoxaluria type 1
|
article |
2020/06/08 |
|
The coronavirus pandemic claims another victim: Medical research for deadly rare diseases
|
article |
2020/06/07 |
|
Researchers develop language test for people with Fragile X syndrome
|
article |
2020/06/01 |
|
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
|
article |
2020/05/30 |
|
5 Things You Should Know About Stiff-Person Syndrome
|
article |
2020/05/30 |
|
Mindfulness training shows promise for people with MS
|
article |
2020/05/26 |
|
Rare Disease Doctors Are Struggling. Here's 3 Ways Patients and Families Can Help.
|
article |
2020/05/24 |
|
When I Had No Choice But to Go to the Hospital During COVID-19 for My Rare Disease
|
article |
2020/05/24 |
|
In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical Relief Program
|
article |
2020/05/17 |
|
NIH-supported research survey to examine impact of COVID-19 on rare diseases community
|
article |
2020/05/10 |
|
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide
|
article |
2020/05/10 |
|
COVID-19 pandemic hinders access to care for patients with rare diseases
|
article |
2020/05/10 |
|
Commentary: What those with rare diseases want us to consider
|
article |
2020/05/05 |
|
Rare Illness in Children May Be Related to Coronavirus, European Doctors Are Warned
|
article |
2020/05/05 |
|
Immune Thrombocytopenic Purpura in a Patient with Covid-19
|
article |
2020/04/30 |
|
Guillain–Barré Syndrome Associated with SARS-CoV-2
|
article |
2020/04/25 |
|
European Commission approves nintedanib for the treatment of systemic sclerosis-associated interstitial lung disease (SSc-ILD)
|
article |
2020/04/25 |
|
Russia’s Coronavirus Outbreak Jeopardizes Care for Patients With Rare Diseases
|
article |
2020/04/25 |
|
Chiesi Global Rare Diseases Recognizes Fabry Disease Awareness Month and Launches “Fabry Focus on Health” Educational Series to Support Patients and Caregivers During COVID-19 Pandemic
|
article |
2020/04/25 |
|
NORD Launches Financial Assistance Program for Rare Disease Community Members Impacted by COVID-19
|
article |
2020/04/25 |
|
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide
|
article |
2020/04/19 |
|
Inspirational boy battling rare disease returns from hospital after 'life-saving' surgery
|
article |
2020/04/19 |
|
Who's Equipped to Deal With Pandemic Worries? Patients With Rare Diseases, Some Say
|
article |
2020/04/19 |
|
RDMD Lands $14M to Expand Reach of Rare Disease Research Platform
|
article |
2020/04/19 |
|
Helping Patients With Rare Dieases During the Coronavirus Pandemic
|
video |
2020/04/12 |
|
Dublin teen's film on rare disease chosen for international festival
|
article |
2020/04/12 |
|
FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease
|
article |
2020/04/12 |
|
A Genetic Test For A Microscopic Problem Came With A Jumbo Price Tag
|
article |
2020/04/05 |
|
COVID-19 in a patient with systemic sclerosis treated with tocilizumab for SSc-ILD
|
article |
2020/04/02 |
|
Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies
|
article |
2020/03/29 |
|
Thank You for Your COVID-19 Response — from a Rare Disease Patient
|
article |
2020/03/28 |
|
Toronto family of child with ultra-rare disease urges public to take COVID-19 seriously
|
article |
2020/03/28 |
|
Antioch teen with rare disease won’t let COVID-19 stop her
|
article |
2020/03/21 |
|
5 Things You Should Know About Vasculitis
|
article |
2020/03/21 |
|
How People With Weakened Immune Systems Are Navigating The Coronavirus
|
podcast |
2020/03/21 |
|
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
|
article |
2020/03/21 |
|
What to Do If Your Child Is Diagnosed With a Rare Disease
|
article |
2020/03/21 |
|
‘My immune system is very susceptible’: Woman with rare disease wants you to take COVID-19 seriously
|
video |
2020/03/15 |
|
How Can We Improve the Diagnosis and Treatment of Rare Diseases?
|
article |
2020/03/15 |
|
The Importance of Research on Rare Diseases
|
article |
2020/03/15 |
|
The Bad Economics of the U.S. Health Care System Shows Up Starkly in Its Approach to Rare Diseases
|
article |
2020/03/15 |
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Genomics in healthcare: How systems are using the data and whether there is a cause for concern
|
article |
2020/03/12 |
|
Testing for rare diseases at birth may have spared our girl a life of suffering, says mother of five-year-old who has degenerative brain condition
|
article |
2020/03/04 |
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She was asked to speak about her two ‘ultrarare’ diseases. She did, while also telling of a rare place.
|
article |
2020/03/04 |
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The Challenges Of Combating Rare Diseases - And Five Innovations Making A Real Difference
|
article |
2020/03/04 |
|
Evans syndrome: Everything you need to know
|
article |
2020/02/23 |
|
Mercer Island family raises awareness for rare, undiagnosed diseases
|
article |
2020/02/23 |
|
A Rare Human Disease Has Been Found in Dinosaur Bones, Could it Lead to a Treatment?
|
article |
2020/02/19 |
|
Gene therapy to halt rare form of sight loss
|
article |
2020/02/17 |
|
Meet the man who invented Viagra: He's fighting rare diseases with artificial intelligence to treat patients
|
video |
2020/02/09 |
|
Medicine by machine: Is A.I. the cure for the world’s ailing drug industry?
|
article |
2020/02/09 |
|
Rare Neurological Disease Treatment Market to Record CAGR of 8.7% Increase in Revenue by 2026
|
article |
2020/02/09 |
|
Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases
|
article |
2020/02/09 |
|
Chiesi creates Boston rare disease unit ahead of Fabry launch
|
article |
2020/02/09 |
|
Nature Outlook: RNA Therapies
|
article |
2020/02/02 |
|
Sanofi Sees Positive Results in Two Clinical Trials for Niemann-Pick Disease
|
article |
2020/02/02 |
|
He's the oldest-known man with rare Proteus syndrome and he hopes to help find a cure
|
article |
2020/02/02 |
|
The Conundrum Of Investing In Cardiovascular Versus Rare Diseases R&D
|
article |
2020/01/26 |
|
Randall Rutta: Discrimination against people with rare diseases
|
article |
2020/01/26 |
|
Apple to Participate in Meeting Advocating for Better Patient Access to Health Info
|
article |
2020/01/26 |
|
4 deeply personal stories about the impact of genetic testing
|
article |
2020/01/26 |
|
Access And Actionability Are Key For Genetic Testing And Precision Medicine
|
article |
2020/01/18 |
|
FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development
|
article |
2020/01/18 |
|
Drew Brees gives emotional speech as Steve Gleason receives Congressional Gold Medal
|
article |
2020/01/18 |
|
What you need to know about HLH, the rare disease linked to ESPN reporter Edward Aschoff's death
|
article |
2020/01/13 |
|
'Revolutionary' DNA test can detect thousands of rare diseases in children
|
article |
2020/01/03 |
|
Toronto parents of baby needing $2.8M drug turn to Swiss drugmaker's dose lottery as a last resort
|
article |
2020/01/01 |
|
A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment
|
podcast |
2019/12/28 |
|
7 Pro Tips to Help Deal With the Anxiety of Rare Disease Parenting
|
article |
2019/12/28 |
|
27 Years of Humanitarian Programs in Vietnam
|
article |
2019/12/22 |
|
A Harvard geneticist is creating a dating app that matches users based on DNA, and people are worried it's eugenics
|
article |
2019/12/15 |
|
Rare Disease Q&A: What Rare Diseases Are and Why That Matters
|
article |
2019/12/15 |
|
Website tackles an all-too-common problem with rare diseases: A shortage of information
|
article |
2019/12/15 |
|
Scientists suggest new solution to the rare-disease problem
|
article |
2019/12/15 |
|
ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa
|
article |
2019/12/12 |
|
Angelman Advocate Launches ‘Combined Brain’ Rare Disease Consortium
|
article |
2019/12/12 |
|
FDA approves first treatment for inherited rare disease
|
article |
2019/12/12 |
|
GBT Snags FDA Approval for First-of-its-Kind Sickle Cell Disease Treatment
|
article |
2019/11/27 |
|
Most ER Staff Don't Understand Rare Disease, So This Is How I Advocate for Myself
|
article |
2019/11/24 |
|
Doctors said boy wouldn't live past his second birthday. His town just threw a parade for his third
|
article |
2019/11/24 |
|
FDA Approves REBLOZYL® (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions
|
article |
2019/11/17 |
|
Rare diseases more common than we think
|
article |
2019/11/10 |
|
U.S. patients with rare diseases go to the doctor an average of 8 times to find answers
|
video |
2019/11/05 |
|
When Your Child With a Rare Disease Doesn’t Feel Brave
|
article |
2019/10/28 |
|
Why We Decided to Start the First Rare Disease Film Festival
|
article |
2019/10/28 |
|
7 rare diseases a rural Wisconsin doctor found in his Amish and Mennonite patients
|
article |
2019/10/19 |
|
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
|
article |
2019/10/13 |
|
FDA approves first treatment to increase pain-free light exposure in patients with a rare disorder
|
article |
2019/10/13 |
|
NIH funding bolsters rare diseases research collaborations
|
article |
2019/10/05 |
|
ICER discriminates against people with rare diseases
|
article |
2019/10/05 |
|
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels
|
article |
2019/09/29 |
|
Infographic: Which Rare Diseases Are The Most Common?
|
article |
2019/09/22 |
|
This med student was given last rites before finding a treatment that saved his life. His method could help millions
|
article |
2019/09/15 |
|
Commercialization Imperatives for Success in Rare Diseases
|
article |
2019/09/15 |
|
Priscilla Chan talks her new 'Rare As One' initiative to combat rare diseases
|
video |
2019/09/09 |
|
My life with face blindness
|
article |
2019/09/01 |
|
Boy's Severe Headaches Lead to Rare Eye Disease Diagnosis
|
article |
2019/09/01 |
|
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
|
article |
2019/09/01 |
|
Dr. Google' Helps Some Patients Diagnose a Rare Disease
|
article |
2019/09/01 |
|
This Penn Professor Almost Died From a Rare Disease Four Times
|
article |
2019/09/01 |
|
Boy, three, who was diagnosed with a rare neurological condition which affects just one in a BILLION, is able to walk for the first time after his parents raised £16k for a 'life-changing' machine
|
article |
2019/08/25 |
|
13 Things ER Staff Should Know About People With Rare Diseases
|
article |
2019/08/25 |
|
New Study Proves the Internet Can Help You Get a Rare Disease Diagnosis
|
article |
2019/08/20 |
|
Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome
|
article |
2019/08/17 |
|
ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)
|
article |
2019/08/17 |
|
FDA approves treatment for patients with rare bone marrow disorder
|
article |
2019/08/17 |
|
Internet can be valuable tool for people with undiagnosed rare disorders
|
article |
2019/08/11 |
|
Have 12 friends? You probably know someone with a rare disease
|
article |
2019/08/11 |
|
These two Raytown women have a rare disease. Why don’t more doctors want to help?
|
video |
2019/08/11 |
|
Rare diseases are not uncommon, and may be the mother lode for the world’s pharmaceutical producers and researchers
|
article |
2019/08/05 |
|
Denver family fundraising to help toddler suffering from rare disease
|
video |
2019/08/05 |
|
European initiative targets diagnosis, treatment of rare diseases
|
article |
2019/08/05 |
|
FDA approves pexidartinib for tenosynovial giant cell tumor
|
article |
2019/08/02 |
|
In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder
|
podcast |
2019/07/30 |
|
This college dropout was bedridden for 11 years. Then he invented a surgery and cured himself
|
article |
2019/07/28 |
|
Rare diseases prompted care in ancient times
|
article |
2019/07/28 |
|
Apic Bio’s APB-102 Receives Orphan Drug Designation from the FDA for the Treatment of Genetic SOD1 ALS
|
article |
2019/07/23 |
|
Boehringer Ingelheim Gambles $2.62 Billion on Two Fibrotic Disease Deals
|
article |
2019/07/23 |
|
New Hope for Rare Mitochondrial Diseases: An Interview With Minovia Therapeutics’ Natalie Yivgi Ohana
|
article |
2019/07/23 |
|
Stranger's bone marrow donation saves 1-year-old baby girl with rare 'bubble boy' disease
|
article |
2019/07/13 |
|
Drug shortage threatens health of O'Fallon man
|
video |
2019/07/09 |
|
A Xi'an girl's escape from early death sheds light on China's arduous journey to make drugs affordable for rare diseases
|
article |
2019/07/09 |
|
Rare diseases are not uncommon, and may be the mother lode for the world's pharmaceutical producers and researchers
|
article |
2019/07/09 |
|
FDA approves first treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system
|
article |
2019/06/30 |
|
China is picking up the fight against rare diseases
|
article |
2019/06/30 |
|
25-year-old faces rare form of ALS that took her twin, but experimental drug offers hope
|
article |
2019/06/22 |
|
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
|
article |
2019/06/22 |
|
5 Questions: Stephen Montgomery on RNA’s role in diagnosing rare diseases
|
article |
2019/06/22 |
|
When a Rare Mutation Causes a Rare Disease: Jacob’s Story
|
article |
2019/06/22 |
|
When a Rare Mutation Causes a Rare Disease: Jacob’s Story
|
article |
2019/06/22 |
|
Drug Discovery for Rare Diseases
|
article |
2019/06/22 |
|
Denali's Hunter Syndrome Candidate Gets Orphan Drug Status
|
article |
2019/06/16 |
|
Chan Zuckerberg Initiative launches new program to support rare disease patients
|
article |
2019/06/16 |
|
How artificial intelligence can help detect rare diseases
|
article |
2019/06/16 |
|
When I Developed the Disease My Father Was Researching
|
article |
2019/06/02 |
|
California parents hope for miracle after baby diagnosed with rare, fatal genetic disorder
|
article |
2019/05/19 |
|
I have a rare disease. This is my hope for the future of medicine
|
article |
2019/05/19 |
|
Hearing loss and rare diseases: What's the connection?
|
article |
2019/05/19 |
|
FDA poised to approve $5 MILLION gene therapy to treat muscular atrophy - making it the most expensive drug on the planet
|
article |
2019/05/19 |
|
FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder
|
article |
2019/05/12 |
|
They don't know if their children will ever walk or talk. But finding other families online has given them hope.
|
article |
2019/05/12 |
|
Rare Disease R&D Investments Likely To Grow In Biopharma
|
article |
2019/05/08 |
|
An experimental test may help confirm cases of chronic fatigue syndrome
|
article |
2019/05/08 |
|
PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights
|
article |
2019/05/08 |
|
Potential Gene Therapy Safely Restores FVIII Clotting Factor in Animals and Human Cells, ASC Therapeutics Reports
|
article |
2019/05/08 |
|
Montreal doctors’ 'Hail Mary’ treatment could save babies' lives from most common of all rare diseases
|
article |
2019/05/08 |
|
U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease
|
article |
2019/05/08 |
|
An experimental test may help confirm cases of chronic fatigue syndrome
|
article |
2019/05/02 |
|
Rare Disease R&D Investments Likely To Grow In Biopharma
|
article |
2019/05/02 |
|
Designer Microbes: a Potential Living Cure for Crohn's and Phenylketonuria
|
article |
2019/04/21 |
|
St. Jude gene therapy cures babies with 'bubble boy' disease
|
article |
2019/04/20 |
|
How UK startup Mendelian hopes to use machine learning to diagnose rare diseases
|
article |
2019/04/20 |
|
Mom Is Tirelessly Raising Awareness About Her Daughter's Rare, Life-Threatening Disease
|
article |
2019/04/20 |
|
Texas Baby Born Without Skin Leaves Doctors Scrambling for Answers and Battling to Save His Life
|
article |
2019/04/20 |
|
Opinion: Value-based agreements could disrupt how we pay for new therapies
|
article |
2019/04/12 |
|
Rare diseases in China, their high treatment costs, and the people publicising patients’ plight
|
article |
2019/04/12 |
|
Rare Gut Condition A Model For Study Of Genetic Diseases
|
article |
2019/04/12 |
|
Rare Diseases: A Psychiatric Concern?
|
article |
2019/04/04 |
|
What I’ve Learned Since My Diagnosis with a Rare Blood Cancer
|
article |
2019/04/04 |
|
Gene therapy targets sickle-cell disease
|
article |
2019/04/04 |
|
Change the genes to fix the skin
|
article |
2019/04/04 |
|
Experimental Therapy Gives Children With Genetic Disorders A New Chance At Life
|
video |
2019/03/25 |
|
Alexion and Affibody Announce Partnership to Co-Develop Anti-FcRn Affibody® Molecule
|
article |
2019/03/25 |
|
Pfizer Steps Further In The Door For Gene Therapies
|
article |
2019/03/25 |
|
CMN: 'For someone to see the way I look as art is amazing'
|
article |
2019/03/18 |
|
FDA is Working to Bridge Gaps and Meet Needs for Rare Disease Product Development
|
article |
2019/03/18 |
|
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
|
article |
2019/03/18 |
|
Could gene therapy cure sickle cell anemia?
|
video |
2019/03/11 |
|
Alnylam's Givosiran Looks Positive Overall in Phase III Trial for Rare Disease
|
article |
2019/03/07 |
|
Life for a child with a rare disease is a grueling roller coaster ride: A mother explains
|
article |
2019/03/06 |
|
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
|
article |
2019/03/06 |
|
Mom Blazes Path to Cure for Son’s Rare Disease
|
article |
2019/03/06 |
|
Rich and Caitlin Hill pledge $575,000 to MGH for genetic disease research
|
article |
2019/03/01 |
|
Finding New Uses For Old Drugs - A Lifeline For Rare Disease Patients
|
article |
2019/03/01 |
|
Healx Pushing New Drug Development Model in Rare Diseases with Barth Syndrome Foundation
|
article |
2019/03/01 |
|
The rare disease challenge meeting
|
article |
2019/02/19 |
|
I have a rare disease. Research on such illnesses can provide insights into more common ones.
|
article |
2019/02/19 |
|
Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
|
article |
2019/02/19 |
|
Once free, drug for rare disease now being sold for $375K a year
|
video |
2019/02/11 |
|
Sanofi Snags FDA Approval for Rare Blood Disorder
|
article |
2019/02/10 |
|
Could This Radical New Approach to Alzheimer’s Lead to a Breakthrough?
|
article |
2019/02/10 |
|
First US attempt to cure a rare disease with genome editing fails miserably
|
article |
2019/02/10 |
|
These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.
|
article |
2019/02/02 |
|
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
|
article |
2019/02/02 |
|
Lysosomal Acid Lipase Deficiency Financial Assistance Program Launched by The Assistance Fund
|
article |
2019/01/26 |
|
Advances in inborn errors of metabolism
|
article |
2019/01/26 |
|
FDA's Banner Year For Approvals: Will Market Access To New Drugs Follow Suit?
|
article |
2019/01/26 |
|
Moyamoya: The rare disease you've never heard of
|
video |
2019/01/20 |
|
East Bay family launches national campaign to save life of 5-year-old who needs bone marrow transplant
|
article |
2019/01/20 |
|
AI technology can identify genetic diseases by looking at your face, study says
|
article |
2019/01/15 |
|
Podcast Extra: The quest for a rare disease treatment
|
podcast |
2019/01/15 |
|
In developing new drugs for rare diseases, this non-profit steps in when pharmaceutical companies won't
|
article |
2019/01/15 |
|
Genetic screening for newborns yields some answers, more questions
|
article |
2019/01/05 |
|
Finding Answers: Statesville woman's rare disease needs rare treatment
|
article |
2019/01/05 |
|
Medical Detectives: The Last Hope For Families Coping With Rare Diseases
|
article |
2019/01/05 |
|
15-month-old Denver girl fighting rare disease, raising awareness for treatments and a cure
|
article |
2019/01/05 |
|
Researchers find a potential new combination therapy against a rare disease
|
article |
2019/01/05 |
|
FDA approves new treatment for rare disease of the bone marrow and blood
|
article |
2019/01/05 |
|
Exclusions: Adding Complexity for Patients With Rare Diseases
|
article |
2018/12/25 |
|
Sangamo Announces 16 Week Clinical Results Including Reductions In Glycosaminoglycans In Phase 1/2 Trial Evaluating SB-913, A Zinc Finger Nuclease Genome Editing Treatment For MPS II (Hunter Syndrome)
|
article |
2018/12/25 |
|
Live bacteria deliver crucial enzymes straight to the gut
|
article |
2018/12/22 |
|
Axovant Licenses Two Gene Therapies Aimed at Tay-Sachs and Sandhoff Disease
|
article |
2018/12/19 |
|
Medical Detectives: The Last Hope for Families Coping with Rare Diseases
|
article |
2018/12/19 |
|
Undiagnosed rare diseases costing NHS over £3bn, study shows
|
article |
2018/12/19 |
|
Navigating the Rare Disease Space: Insights from a Danish CEO
|
article |
2018/12/19 |
|
National Gathering of the Rare Disease Community and Health Professionals Launched with 2019 Living Rare, Living Stronger | NORD Patient & Family Forum
|
article |
2018/12/09 |
|
Man diagnosed with rare disease takes 2 life-changing steps
|
article |
2018/12/09 |
|
Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research
|
article |
2018/12/09 |
|
Dansville teen overcomes rare disease, returns to dance
|
article |
2018/12/09 |
|
Rare Youth Project: Can you empower the next generation of rare disease advocates?
|
article |
2018/11/28 |
|
Dublin Boy With Rare Disease Gets Special Bike
|
article |
2018/11/28 |
|
Cheers, tears as Staten Island teen with rare disease receives new home
|
article |
2018/11/28 |
|
FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease
|
article |
2018/11/28 |
|
Vivek Ramaswamy's Latest "Vant": Aruvant to Focus on Sickle Cell and Beta-Thalassemia
|
article |
2018/11/28 |
|
Women with a Rare Disease Take Control of Their Destiny
|
article |
2018/11/18 |
|
How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025
|
article |
2018/11/18 |
|
Ravenna boy battles rare disease
|
article |
2018/11/18 |
|
Rare disease acromegaly finds an Ottawa champion
|
article |
2018/11/18 |
|
Students transform school into Hogwarts to surprise classmate fighting rare disease
|
article |
2018/11/18 |
|
Local Hero with rare disease to be honored at 2018 MG Walk at Bear Creek Pioneers Park
|
article |
2018/11/18 |
|
Benefit held for local woman battling rare disease
|
video |
2018/11/18 |
|
Rare diseases require radical new funding methods
|
article |
2018/11/18 |
|
Patients: The Catalysts Behind Rare Disease Research & Drug Development
|
article |
2018/10/28 |
|
How FDA's Breakthrough Therapy Designation Program Changed the Rare Disease Space
|
article |
2018/10/28 |
|
Updated Study Analyzes Use and Cost of Orphan Drugs
|
article |
2018/10/28 |
|
Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease
|
article |
2018/10/28 |
|
Nature Outlook: Huntington's Disease
|
article |
2018/10/28 |
|
Nature Outlook: Huntington's Disease
|
article |
2018/10/28 |
|
Rare eye condition was behind da Vinci's genius
|
article |
2018/10/21 |
|
Local family shares their journey battling rare disease
|
article |
2018/10/19 |
|
Summerside boy with rare disease gets his wish to visit Legoland in Florida
|
article |
2018/10/19 |
|
A rare-disease entrepreneur follows an unconventional drug development path
|
article |
2018/10/19 |
|
Addressing Diagnosis Challenges in the Rare Disease Space
|
article |
2018/10/19 |
|
First Signs of Huntington's Disease Detectable Via a New Blood Test
|
article |
2018/10/17 |
|
Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial
|
article |
2018/10/16 |
|
Rare Disease Trials Require Patient Insights And Input
|
article |
2018/10/16 |
|
UPCOMING EVENT: The Amyloidosis Foundation's Annual 'Run for Your Life'
|
webpage |
2018/10/13 |
|
Exclusions: Adding Complexity for Patients with Rare Diseases
|
article |
2018/10/10 |
|
Exercise may slow cognitive decline in people with rare Alzheimer's disease
|
article |
2018/10/05 |
|
13-Year-Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food
|
article |
2018/10/04 |
|
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
|
article |
2018/10/03 |
|
One in 67 Hongkongers suffers from a rare disease, study finds
|
article |
2018/10/03 |
|
What to Do When You’re Diagnosed with a Rare Disease
|
article |
2018/10/01 |
|
Rare Pediatric Disease Designation Granted to Stargardt Disease Treatment, LBS-008
|
article |
2018/09/30 |
|
Dubai Science Park Raises Focus on Rare Diseases During Expert Panel
|
article |
2018/09/30 |
|
UPCOMING VIRTUAL EVENT: The 2018 Rare Fair
|
webpage |
2018/09/28 |
|
FDA Awarding Grants for Research into Rare Diseases
|
article |
2018/09/26 |
|
One in 67 Hongkongers suffers from a rare disease, study finds
|
article |
2018/09/25 |
|
FDA Awarding Grants for Research into Rare Diseases
|
article |
2018/09/25 |
|
Mitochondrial Disease: 13 Year Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food
|
article |
2018/09/25 |
|
Autosomal Dominant Alzheimer's Disease: Exercise may slow cognitive decline in people with rare Alzheimer's disease
|
article |
2018/09/25 |
|
Medical foods are vital to treat rare diseases — but some insurers won't pay
|
article |
2018/09/25 |
|
How a Collaborative Ecosystem is Driving the Development of Successful Therapies in Rare Disease Research
|
article |
2018/09/11 |
|
Cystinosis: Firelight Fund to help local family as child battles rare disease
|
article |
2018/09/11 |
|
Developing RNA Medicines for Rare Genetic Diseases
|
article |
2018/09/11 |
|
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases
|
article |
2018/09/10 |
|
Lack of treatment options for rare diseases prompts call for change
|
article |
2018/09/10 |
|
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases
|
article |
2018/09/10 |
|
Ehlers-Danlos Syndrome: Madera boy with rare disease using YouTube to raise awareness
|
article |
2018/09/06 |
|
Med student uses 'Zebra Alphabet' to bring awareness to rare diseases
|
article |
2018/09/03 |
|
Fanconi Anemia: Jimbo Fisher spreads awareness of son's blood disease through nonprofit
|
article |
2018/08/31 |
|
Epidermolysis Bullosa: 'My skin tears and blisters at the slightest touch, but it won't stop me from modelling'
|
article |
2018/08/30 |
|
Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others
|
article |
2018/08/27 |
|
Guillain-Barre Syndrome: What we know about the disease Travis Frederick is fighting
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article |
2018/08/25 |
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Dravet Syndrome: Community rallies around 3-year old battling rare disorder
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article |
2018/08/25 |
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Hereditary Angioedema: Shire wins FDA approval for potential blockbuster rare disease drug
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article |
2018/08/24 |
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Biallelic HMBS Deficiency: 'Kure 4 Kam': Parents navigate medical maze so 1-year-old son might withstand rare disease
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article |
2018/08/24 |
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Batten Disease: Norfolk Tides help Virginia Beach teen with rare disease
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article |
2018/08/21 |
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With $3m in funding, tech firm looks to drive drug development using EHR data
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article |
2018/08/21 |
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Acute Necrotizing Encephalopathy: Moree community supports two-year-old Paige Thomas as she fights rare brain disease
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article |
2018/08/21 |
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Familial Chylomicronemia Syndrome: Ultra-rare disease patients find support in connectedness
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article |
2018/08/21 |
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Hurler Syndrome: World record attempt aims to help local boy battle rare disease
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article |
2018/08/21 |
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L1 Cam Syndrome: $1K Needed To Help Dublin Boy With Rare Disease In Need Of Bike
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article |
2018/08/20 |
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Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech
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article |
2018/08/19 |
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Niemann-Pick Disease: Reno twins with rare, fatal disease will be on CNN show "Something's Killing Me"
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article |
2018/08/17 |
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Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant
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article |
2018/08/17 |
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California group helps people with rare diseases aim for new heights
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article |
2018/08/16 |
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Epidermodysplasia Verruciformis: New Link Found Between A Rare Skin Disease and Beta-HPV
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article |
2018/08/16 |
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Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease
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article |
2018/08/15 |
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Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk
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article |
2018/08/14 |
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Fabry Disease: FDA Approves Galafold for Rare Genetic Disorder, Fabry Disease
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article |
2018/08/14 |
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Hunter Syndrome: Talking about rare disease
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article |
2018/08/13 |
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hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
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article |
2018/08/10 |
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Sarcoidosis: Local nurse leads support group for rare disease
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article |
2018/08/05 |
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Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest
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article |
2018/08/02 |
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Lamellar Ichthyosis: Mum says her baby girl’s skin is like ‘petting a SNAKE’ due to rare condition that means she has to bathe in bleach
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article |
2018/08/02 |
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Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria
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article |
2018/08/01 |
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Hermansky-Pudlak Syndrome: MN man battles rare genetic disorder
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article |
2018/07/31 |
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Familial Amyloid Polyneuropathy: What caused this man's scalloped pupil?
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article |
2018/07/31 |
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PODCAST: Rare Unplugged: Episode 1
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podcast |
2018/07/30 |
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Stiff Skin Syndrome: 'I email 100 doctors a day': Mother of boy, 12, with rare terminal disease that is turning him 'to STONE' describes her desperate attempts to find a cure
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article |
2018/07/30 |
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Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington
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article |
2018/07/29 |
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FDA Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC
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article |
2018/07/29 |
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Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun
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article |
2018/07/29 |
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AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu
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article |
2018/07/26 |
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Adult Polyglucosan Body Disease: Film series pick highlights rare disease - Jewish Exponent
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article |
2018/07/25 |
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Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan
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article |
2018/07/24 |
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North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte
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article |
2018/07/24 |
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Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia
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article |
2018/07/21 |
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Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com
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podcast |
2018/07/19 |
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Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat
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article |
2018/07/19 |
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MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte
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article |
2018/07/18 |
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Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People
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article |
2018/07/18 |
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Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana
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article |
2018/07/17 |
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CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo
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article |
2018/07/16 |
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CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com
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article |
2018/07/12 |
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Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland
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article |
2018/07/12 |
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Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco
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article |
2018/07/12 |
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Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report
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article |
2018/07/10 |
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Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org
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article |
2018/03/07 |
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PODCAST: Rare In Common: Hanging Out With Zebras: Life with a genetic mutation so rare it has never been reported before - RareInCommon.com
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podcast |
2018/02/28 |
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VIDEO: Rare In Common
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video |
2017/02/27 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes
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article |
2016/07/07 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes
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article |
2016/06/27 |
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VIDEO: Super Power Baby Project - TEDxAuckland
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video |
2014/10/09 |
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September, 2009 | Patient Voices: Rare Diseases - The New York Times
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article |
2009/09/01 |
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