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Title Category News Date Link
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
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