News Links

Title	Category	News Date
Rare vs common diseases: key differences in educational approaches	article	2024/12/19
Rare diseases and health equity	article	2024/12/19
Can AI Improve Diagnosis of Rare Diseases?	article	2024/12/19
He diagnosed his rare disease using Google. Now he hopes AI can do the same for others	article	2024/12/19
A 10-year-old Parker girl’s disease is one in a million, and advancements in her lifetime made all the difference	article	2024/12/19
For kids with rare genetic disorders, customized CRISPR treatments offer hope	article	2024/12/14
Rare disease diagnosis strengthens bond between twin sisters: ‘Doing our best’	article	2024/12/06
New insights into Niemann-Pick type C: A form of childhood dementia	article	2024/12/06
Hemophagocytic Lymphohistiocytosis in two Romanian Young Infants	article	2024/12/06
Newborn screening by genome sequencing shown to be safe and effective in two clinical studies	article	2024/12/06
Cherokee Nation hosts inaugural Rare Disease Summit	article	2024/11/30
US FDA declines to approve Applied Therapeutics’ genetic disease drug	article	2024/11/30
Scientists find cause of rare devastating, autoimmune disease in Puerto Ricans—but also find potential treatment	article	2024/11/30
Stone man disease: A rare condition that causes a person to grow a second skeleton	article	2024/11/30
Sage Therapeutics scraps development of rare disease drug after it fails mid-stage study	article	2024/11/21
Butterfly disease: A disorder that makes skin as delicate as butterfly wings	article	2024/11/21
ROI and Rare Disease: Retooling the ‘Gene’ Value Machine	article	2024/11/15
CHEO Hopes to Share World-First AI Algorithm to Diagnose Rare Disease	article	2024/11/15
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub	article	2024/11/08
One gene provides diagnoses for 30 patients whose condition was unexplained for years	article	2024/11/08
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?	article	2024/11/08
Rare disease initiative aims to speed diagnoses and treatment in Latin America	article	2024/11/01
Improving the recipe for gene therapy: New treatment for Gaucher disease shows promise	article	2024/10/18
Family selling dream home to fund life-saving treatment for 5-year-old daughter	article	2024/10/18
Impossible' FDA adcomm ends in favor of Stealth’s ultra-rare disease med	article	2024/10/12
Sentynl Therapeutics: Next Generation Sequencing (NGS) and Rare Disease Diagnostics	article	2024/10/12
TikTok star Taylor Rousseau Grigg died after complications from Addison's disease, family says. What to know about the rare condition.	article	2024/10/12
7-Year-Old with Rare Disease — 'Basically Childhood Dementia' — Needs $172,000 Therapy Every 2 Weeks	article	2024/10/12
RARE's Wilson Disease Candidate Betters Standard Therapy in Study	article	2024/10/04
Sanofi Drug for Rare Blood Disease Is Heading to Recordati for $825M	article	2024/10/04
Why your chance of having a rare disease depends on where you live	article	2024/10/04
NHS launches study of newborn blood test to detect 200 rare conditions	article	2024/10/04
The Future of Rare Disease Treatment with Precision Medicine	article	2024/10/04
Our Rare Disease Network Goes Global	article	2024/10/04
7-year-old girl with rare disease forced to hide from sun, cover every inch of skin: ‘Complete gut punch’	article	2024/09/27
I knew something was wrong with my newborn but doctors ignored me- months later my baby was diagnosed with a rare disease	article	2024/09/27
Using AI to repurpose existing drugs for treatment of rare diseases	article	2024/09/27
Rare Diagnoses Change People’s Perception of Medical Risk	article	2024/09/27
Scientists Discover Gene Responsible for Rare, Inherited Eye Disease, NIH Reports	article	2024/09/27
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy	article	2024/09/20
US FDA approves Zevra's treatment for rare genetic disease	article	2024/09/20
Rare eye disease doesn't stop sports photographer from focusing on his dream	article	2024/09/13
‘My Low Back Pain Turned Out To Be A Rare Inflammatory Disease With No Cure’	article	2024/09/13
There Was No Treatment for His Son’s Rare Disease, so Dad Moves Mountains to Make One for Children Worldwide	article	2024/09/06
Beyond Rare Disease: Wet AMD Gene Therapy Could Reach Millions	article	2024/09/06
RARE Navigator Pilot Program Aims to Eliminate Critical Gaps in Support for Patients and Families Newly Diagnosed with a Rare Disease	article	2024/09/06
EU Court halts Commission’s ban on drug for rare liver disease	article	2024/09/06
Groundbreaking Study by RDDC and NORD: Unveiling Inequities in the Rare Disease Community Through the Voices of Diverse Patients and Caregivers	article	2024/08/29
The most expensive drugs in the US	article	2024/08/29
EEE virus: What is the mosquito-borne disease affecting the US and how deadly is it?	article	2024/08/29
Rare diseases point to connections between metabolism and immunity	article	2024/08/24
What Is Angelman Syndrome? Everything to Know About the Rare Genetic Disorder Colin Farrell’s Son James Has	article	2024/08/10
After 40 years of pain, Erie man diagnosed with life-changing rare disease	article	2024/08/10
Rare Disease-Focused Applied Therapeutics' Lead Candidate Govorestat Has Blockbuster Potential, Analyst Sees 140% Stock Upside	article	2024/08/03
‘This is real’: Newport Beach family seeks to ensure rare disease funding continues	article	2024/08/03
Genetic diagnostics of ultra-rare diseases: Large multicenter study identifies 34 new genetic diseases	article	2024/07/27
Inside the efforts to rescue a rare disease gene therapy	article	2024/07/27
My Uncle Donald Trump Told Me Disabled Americans Like My Son ‘Should Just Die’	article	2024/07/27
Using AI to Improve Detection of Rare Diseases	article	2024/07/19
Ionis (IONS) Completes Enrolment for Rare Disease Drug Study	article	2024/07/19
FDA launches new rare disease innovation hub	article	2024/07/19
‘Crouching Tiger, Hidden Dragon’ star Cheng Pei-pei dead at 78	article	2024/07/19
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
Connecting the Clinical, Regulatory, and Commercial Dots for Rare Diseases	article	2024/07/12
AI-Powered Tool Combs through Electronic Health Records For Faster Diagnoses of Rare Diseases	article	2024/07/06
Left in limbo: When pharma halts rare disease research	article	2024/07/06
Making genome sequencing a first-line test in rare disease	article	2024/06/28
FDA Opens Doors for More Treatments for Rare Diseases through the New START Pilot Program	article	2024/06/28
Gene therapy halts progression of rare genetic condition in young boy	article	2024/06/28
AWS commits $10m for paediatric health and rare diseases research	article	2024/06/28
‘There is no cure for this’: Rare disease causing 6-year-old girl to age rapidly	video	2024/06/28
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate	article	2024/06/23
Whole Genome Sequencing Proven Superior for Identifying Genetic Causes of Rare Diseases	article	2024/06/15
Vertex says Casgevy benefit extends to nearly five years in sickle cell disease	article	2024/06/15
Beyond Biotech - the podcast from Labiotech Developing treatments for rare pediatric diseases	podcast	2024/06/08
Moderna and Myrtelle among those picked for FDA rare disease programme	article	2024/06/08
Denali, Neurogene and more picked by FDA's 'Operation Warp Speed' for rare disease	article	2024/06/08
Moderna's MMA candidate nabs spot in FDA pilot for rare disease drugs	article	2024/06/08
Rare disease's mutation could explain more common conditions	article	2024/06/08
FDA Approves Eculizumab-Aeeb as Interchangeable Biosimilar for 2 Rare Diseases	article	2024/06/02
Existing drug shows promise as treatment for rare genetic disorder	article	2024/06/02
Deadly brain disease ‘doesn’t feel rare’ to this Michigan woman’s family	article	2024/05/27
Rare disease clinical trials	article	2024/05/27
Genetic diagnostic technology a game changer for rare diseases, but ethics concerns linger	article	2024/05/27
Peter Alexander’s Sister Rebecca Recalls Progressive Vision and Hearing Loss Since Age 12 Due to Rare Disease	article	2024/05/18
Alabama will screen newborns for more rare diseases thanks to Foley teen, family and advocates	article	2024/05/18
These congressmen want to modify the Inflation Reduction Act to foster rare disease research	article	2024/05/10
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
Rare disorder causes man to see people's faces as 'demonic'	article	2024/05/10
In Washington DC and Gaza two very different families are united by one very rare disease	article	2024/05/10
Persistence leads to diagnosis of ultra-rare disease	article	2024/05/10
Pilot program improves well-being of families during advanced care planning	article	2024/05/03
AI Might Spot Rare Diseases in Patients Years Earlier	article	2024/05/03
To save lives, the FDA must change how it evaluates rare disease treatments	article	2024/05/03
Imagine living in a 4-foot body that doesn't develop chronic diseases	article	2024/05/03
AI Is Transforming Drug Matching for Cancer, Rare Diseases — Here's How	article	2024/04/27
As more rare disease therapies launch, their prices are rising	article	2024/04/19
Peter Frampton rocks on despite potentially crippling disease: ‘I’m a fighter,’ says the 74-year-old guitar great	article	2024/04/12
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
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
Gene Therapy for Rare Neurodegenerative Disease Shows Promise in Trial	article	2024/03/21
FDA approves new treatment for rare liver disorder	article	2024/03/14
Big league bartenders a hit at annual fundraiser for rare disease	article	2024/03/14
New way for states to cover pricey gene therapies will start with sickle cell disease	article	2024/03/14
When Should a Pediatrician Suspect a Rare Disease?	article	2024/03/14
My daughter has a rare disease. We shouldn't have had to leave the US to save her life.	article	2024/03/09
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
Amy Schumer has been diagnosed with Cushing syndrome. What to know about the rare disorder	article	2024/02/25
‘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
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
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
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
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	article	2021/06/27
Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines	article	2021/06/20
The Loneliness I Feel as the Parent of a Child With a Rare Disease	article	2021/06/20
Columbus mom lost one daughter to a rare disease. A new drug is helping another	video	2021/06/12
KC family worries cost could keep drug from helping son with rare disorder	video	2021/06/12
Raleigh biopharma company hopes to have a new treatment for celiac disease	article	2021/06/05
Unprecedented data sharing driving new rare disease diagnoses in Europe	article	2021/06/05
FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder	article	2021/06/05
The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price	article	2021/06/05
Newer methods may boost gene therapy's use for more diseases	article	2021/06/05
Child fights for her life by taking the 'most expensive drug in the world'	video	2021/05/21
‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access	article	2021/05/21
Las Vegas woman writes children's book to help people with rare disease	video	2021/05/21
Treating Polycythemia Vera	article	2021/05/16
Rare diseases the next target for mRNA therapies	article	2021/05/16
Why I Hate My Kid's Birthday	article	2021/05/16
Moms search for lifesaving bone marrow donors for daughters with rare diseases	article	2021/05/16
Rare Disease Companies Unite to Advocate for Life-Changing Therapies	article	2021/05/16
Cystic Fibrosis: One Patient’s Story, Current Treatments and Exciting New Therapies on the Horizon	article	2021/05/16
Gap in care, research and funding for rare blood disorder disproportionately affecting Black Americans	video	2021/05/09
CENTOGENE Discovers Six New Rare Diseases by Leveraging the Strength of Its Bio/Databank	article	2021/05/09
Virtual gala to raise funds, awareness for rare neurodevelopmental disorder	article	2021/05/02
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
CDISC and NORD Partner to Develop Data Standards for Rare Diseases	article	2021/04/24
A new treatment for rare muscular disease	article	2021/04/17
Detecting rare disease: Revealing the methods, motivations, and implications	article	2021/04/17
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US	article	2021/04/17
Opinion: Genome sequencing can lead to life-changing care for infants. California should make it more available	article	2021/04/10
Keenan: Reseachers are 'in this together' to solve rare diseases	article	2021/04/10
Ducks’ Brady Breeze ‘repping for rare diseases’ at pro day	video	2021/04/03
Software engineer bets on technology to help speed rare disease treatments	article	2021/04/03
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
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
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
7,000 challenges: The basis and burden of rare diseases	article	2021/02/28
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
Guest column: Innovative testing detects rare diseases in critically ill infants	article	2021/02/21
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
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
She was asked to speak about her two ‘ultrarare’ diseases. She did, while also telling of a rare place.	article	2020/03/04
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	article	2018/08/25
Dravet Syndrome: Community rallies around 3-year old battling rare disorder	article	2018/08/25
Hereditary Angioedema: Shire wins FDA approval for potential blockbuster rare disease drug	article	2018/08/24
Biallelic HMBS Deficiency: 'Kure 4 Kam': Parents navigate medical maze so 1-year-old son might withstand rare disease	article	2018/08/24
Batten Disease: Norfolk Tides help Virginia Beach teen with rare disease	article	2018/08/21
With $3m in funding, tech firm looks to drive drug development using EHR data	article	2018/08/21
Acute Necrotizing Encephalopathy: Moree community supports two-year-old Paige Thomas as she fights rare brain disease	article	2018/08/21
Familial Chylomicronemia Syndrome: Ultra-rare disease patients find support in connectedness	article	2018/08/21
Hurler Syndrome: World record attempt aims to help local boy battle rare disease	article	2018/08/21
L1 Cam Syndrome: $1K Needed To Help Dublin Boy With Rare Disease In Need Of Bike	article	2018/08/20
Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech	article	2018/08/19
Niemann-Pick Disease: Reno twins with rare, fatal disease will be on CNN show "Something's Killing Me"	article	2018/08/17
Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant	article	2018/08/17
California group helps people with rare diseases aim for new heights	article	2018/08/16
Epidermodysplasia Verruciformis: New Link Found Between A Rare Skin Disease and Beta-HPV	article	2018/08/16
Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease	article	2018/08/15
Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk	article	2018/08/14
Fabry Disease: FDA Approves Galafold for Rare Genetic Disorder, Fabry Disease	article	2018/08/14
Hunter Syndrome: Talking about rare disease	article	2018/08/13
hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease	article	2018/08/10
Sarcoidosis: Local nurse leads support group for rare disease	article	2018/08/05
Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest	article	2018/08/02
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	article	2018/08/02
Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria	article	2018/08/01
Hermansky-Pudlak Syndrome: MN man battles rare genetic disorder	article	2018/07/31
Familial Amyloid Polyneuropathy: What caused this man's scalloped pupil?	article	2018/07/31
PODCAST: Rare Unplugged: Episode 1	podcast	2018/07/30
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	article	2018/07/30
Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington	article	2018/07/29
FDA Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC	article	2018/07/29
Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun	article	2018/07/29
AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu	article	2018/07/26
Adult Polyglucosan Body Disease: Film series pick highlights rare disease - Jewish Exponent	article	2018/07/25
Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan	article	2018/07/24
North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte	article	2018/07/24
Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia	article	2018/07/21
Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com	podcast	2018/07/19
Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat	article	2018/07/19
MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte	article	2018/07/18
Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People	article	2018/07/18
Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana	article	2018/07/17
CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo	article	2018/07/16
CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com	article	2018/07/12
Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland	article	2018/07/12
Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco	article	2018/07/12
Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report	article	2018/07/10
Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org	article	2018/03/07
PODCAST: Rare In Common: Hanging Out With Zebras: Life with a genetic mutation so rare it has never been reported before - RareInCommon.com	podcast	2018/02/28
VIDEO: Rare In Common	video	2017/02/27
Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes	article	2016/07/07
Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes	article	2016/06/27
VIDEO: Super Power Baby Project - TEDxAuckland	video	2014/10/09
September, 2009 \| Patient Voices: Rare Diseases - The New York Times	article	2009/09/01