ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa
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article |
2019/12/12 |
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Angelman Advocate Launches ‘Combined Brain’ Rare Disease Consortium
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article |
2019/12/12 |
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FDA approves first treatment for inherited rare disease
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article |
2019/12/12 |
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GBT Snags FDA Approval for First-of-its-Kind Sickle Cell Disease Treatment
|
article |
2019/11/27 |
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Most ER Staff Don't Understand Rare Disease, So This Is How I Advocate for Myself
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article |
2019/11/24 |
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Doctors said boy wouldn't live past his second birthday. His town just threw a parade for his third
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article |
2019/11/24 |
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FDA Approves REBLOZYL® (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions
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article |
2019/11/17 |
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Rare diseases more common than we think
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article |
2019/11/10 |
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U.S. patients with rare diseases go to the doctor an average of 8 times to find answers
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video |
2019/11/05 |
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When Your Child With a Rare Disease Doesn’t Feel Brave
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article |
2019/10/28 |
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Why We Decided to Start the First Rare Disease Film Festival
|
article |
2019/10/28 |
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7 rare diseases a rural Wisconsin doctor found in his Amish and Mennonite patients
|
article |
2019/10/19 |
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FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
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article |
2019/10/13 |
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FDA approves first treatment to increase pain-free light exposure in patients with a rare disorder
|
article |
2019/10/13 |
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NIH funding bolsters rare diseases research collaborations
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article |
2019/10/05 |
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ICER discriminates against people with rare diseases
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article |
2019/10/05 |
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FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels
|
article |
2019/09/29 |
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Infographic: Which Rare Diseases Are The Most Common?
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article |
2019/09/22 |
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This med student was given last rites before finding a treatment that saved his life. His method could help millions
|
article |
2019/09/15 |
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Commercialization Imperatives for Success in Rare Diseases
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article |
2019/09/15 |
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Priscilla Chan talks her new 'Rare As One' initiative to combat rare diseases
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video |
2019/09/09 |
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My life with face blindness
|
article |
2019/09/01 |
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Boy's Severe Headaches Lead to Rare Eye Disease Diagnosis
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article |
2019/09/01 |
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‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
|
article |
2019/09/01 |
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Dr. Google' Helps Some Patients Diagnose a Rare Disease
|
article |
2019/09/01 |
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This Penn Professor Almost Died From a Rare Disease Four Times
|
article |
2019/09/01 |
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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 |
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13 Things ER Staff Should Know About People With Rare Diseases
|
article |
2019/08/25 |
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New Study Proves the Internet Can Help You Get a Rare Disease Diagnosis
|
article |
2019/08/20 |
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Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome
|
article |
2019/08/17 |
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ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)
|
article |
2019/08/17 |
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FDA approves treatment for patients with rare bone marrow disorder
|
article |
2019/08/17 |
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Internet can be valuable tool for people with undiagnosed rare disorders
|
article |
2019/08/11 |
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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 |
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Rare diseases are not uncommon, and may be the mother lode for the world’s pharmaceutical producers and researchers
|
article |
2019/08/05 |
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Denver family fundraising to help toddler suffering from rare disease
|
video |
2019/08/05 |
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European initiative targets diagnosis, treatment of rare diseases
|
article |
2019/08/05 |
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In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder
|
podcast |
2019/07/30 |
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This college dropout was bedridden for 11 years. Then he invented a surgery and cured himself
|
article |
2019/07/28 |
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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 |
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China is picking up the fight against rare diseases
|
article |
2019/06/30 |
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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 |
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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
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article |
2018/07/25 |
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Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan
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article |
2018/07/24 |
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North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte
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article |
2018/07/24 |
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Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia
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article |
2018/07/21 |
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Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com
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podcast |
2018/07/19 |
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Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat
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article |
2018/07/19 |
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MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte
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article |
2018/07/18 |
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Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People
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article |
2018/07/18 |
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Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana
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article |
2018/07/17 |
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CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo
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article |
2018/07/16 |
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CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com
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article |
2018/07/12 |
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Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland
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article |
2018/07/12 |
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Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco
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article |
2018/07/12 |
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Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report
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article |
2018/07/10 |
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Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org
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article |
2018/03/07 |
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PODCAST: Rare In Common: Hanging Out With Zebras: Life with a genetic mutation so rare it has never been reported before - RareInCommon.com
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podcast |
2018/02/28 |
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VIDEO: Rare In Common
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video |
2017/02/27 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes
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article |
2016/07/07 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes
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article |
2016/06/27 |
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VIDEO: Super Power Baby Project - TEDxAuckland
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video |
2014/10/09 |
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September, 2009 | Patient Voices: Rare Diseases - The New York Times
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article |
2009/09/01 |
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