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Title Category News Date Link
‘My immune system is very susceptible’: Woman with rare disease wants you to take COVID-19 seriously video 2020/03/15
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases article 2019/09/01
Women with a Rare Disease Take Control of Their Destiny article 2018/11/18
With $3m in funding, tech firm looks to drive drug development using EHR data article 2018/08/21
Why We Decided to Start the First Rare Disease Film Festival article 2019/10/28
Who's Equipped to Deal With Pandemic Worries? Patients With Rare Diseases, Some Say article 2020/04/19
When Your Child With a Rare Disease Doesn’t Feel Brave article 2019/10/28
When I Had No Choice But to Go to the Hospital During COVID-19 for My Rare Disease article 2020/05/24
When I Developed the Disease My Father Was Researching article 2019/06/02
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
What you need to know about HLH, the rare disease linked to ESPN reporter Edward Aschoff's death article 2020/01/13
What to Do When You’re Diagnosed with a Rare Disease article 2018/10/01
What to Do If Your Child Is Diagnosed With a Rare Disease article 2020/03/21
What My Family and Friends Want You to Know About Mal de Debarquement Syndrome article 2020/07/01
What I’ve Learned Since My Diagnosis with a Rare Blood Cancer article 2019/04/04
Website tackles an all-too-common problem with rare diseases: A shortage of information article 2019/12/15
Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco article 2018/07/12
Voices from the front line in rare disease article 2020/08/02
Vivek Ramaswamy's Latest "Vant": Aruvant to Focus on Sickle Cell and Beta-Thalassemia article 2018/11/28
VIDEO: Super Power Baby Project - TEDxAuckland video 2014/10/09
VIDEO: Rare In Common video 2017/02/27
Updated Study Analyzes Use and Cost of Orphan Drugs article 2018/10/28
UPCOMING VIRTUAL EVENT: The 2018 Rare Fair webpage 2018/09/28
UPCOMING EVENT: The Amyloidosis Foundation's Annual 'Run for Your Life' webpage 2018/10/13
Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest article 2018/08/02
Undiagnosed rare diseases costing NHS over £3bn, study shows article 2018/12/19
Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome article 2019/08/17
U.S. patients with rare diseases go to the doctor an average of 8 times to find answers video 2019/11/05
U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease article 2019/05/08
Two Patient Deaths Halt Audentes’ Gene Therapy Trial article 2020/07/02
Toronto parents of baby needing $2.8M drug turn to Swiss drugmaker's dose lottery as a last resort article 2020/01/01
Toronto family of child with ultra-rare disease urges public to take COVID-19 seriously article 2020/03/28
This Penn Professor Almost Died From a Rare Disease Four Times article 2019/09/01
This med student was given last rites before finding a treatment that saved his life. His method could help millions article 2019/09/15
This college dropout was bedridden for 11 years. Then he invented a surgery and cured himself article 2019/07/28
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
These two Raytown women have a rare disease. Why don’t more doctors want to help? video 2019/08/11
These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them. article 2019/02/02
The rare disease challenge meeting article 2019/02/19
The Importance of Research on Rare Diseases article 2020/03/15
The coronavirus pandemic claims another victim: Medical research for deadly rare diseases article 2020/06/07
The Conundrum Of Investing In Cardiovascular Versus Rare Diseases R&D article 2020/01/26
The Challenges Of Combating Rare Diseases - And Five Innovations Making A Real Difference article 2020/03/04
The Bad Economics of the U.S. Health Care System Shows Up Starkly in Its Approach to Rare Diseases article 2020/03/15
Thank You for Your COVID-19 Response — from a Rare Disease Patient article 2020/03/28
Texas Baby Born Without Skin Leaves Doctors Scrambling for Answers and Battling to Save His Life article 2019/04/20
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
Summerside boy with rare disease gets his wish to visit Legoland in Florida article 2018/10/19
Study reveals biochemical alterations in patients with Lesch-Nyhan disease article 2020/06/10
Students transform school into Hogwarts to surprise classmate fighting rare disease article 2018/11/18
Stranger's bone marrow donation saves 1-year-old baby girl with rare 'bubble boy' disease article 2019/07/13
Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People article 2018/07/18
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
St. Jude gene therapy cures babies with 'bubble boy' disease article 2019/04/20
Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others article 2018/08/27
Sobi and Sanofi Donate up to 500 Million Additional IUs of Clotting Factor to WFH Humanitarian Aid Program article 2020/06/14
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide article 2020/04/19
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide article 2020/05/10
She was asked to speak about her two ‘ultrarare’ diseases. She did, while also telling of a rare place. article 2020/03/04
September, 2009 | Patient Voices: Rare Diseases - The New York Times article 2009/09/01
Scientists suggest new solution to the rare-disease problem article 2019/12/15
Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease article 2018/10/28
Sarcoidosis: Local nurse leads support group for rare disease article 2018/08/05
Sanofi Snags FDA Approval for Rare Blood Disorder article 2019/02/10
Sanofi Sees Positive Results in Two Clinical Trials for Niemann-Pick Disease article 2020/02/02
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
Russia’s Coronavirus Outbreak Jeopardizes Care for Patients With Rare Diseases article 2020/04/25
Rich and Caitlin Hill pledge $575,000 to MGH for genetic disease research article 2019/03/01
Researchers find a potential new combination therapy against a rare disease article 2019/01/05
Researchers develop new therapeutic approach to treat CLN3 Batten disease article 2020/08/02
Researchers develop language test for people with Fragile X syndrome article 2020/06/01
Recordati Rare Diseases Inc. Announces Availability of ISTURISA® (osilodrostat) in the United States article 2020/06/14
RDMD Lands $14M to Expand Reach of Rare Disease Research Platform article 2020/04/19
Ravenna boy battles rare disease article 2018/11/18
Rare Youth Project: Can you empower the next generation of rare disease advocates? article 2018/11/28
Rare Pediatric Disease Designation Granted to Stargardt Disease Treatment, LBS-008 article 2018/09/30
Rare Neurological Disease Treatment Market to Record CAGR of 8.7% Increase in Revenue by 2026 article 2020/02/09
Rare Illness in Children May Be Related to Coronavirus, European Doctors Are Warned article 2020/05/05
Rare Gut Condition A Model For Study Of Genetic Diseases article 2019/04/12
Rare eye condition was behind da Vinci's genius article 2018/10/21
Rare Diseases: A Psychiatric Concern? article 2019/04/04
Rare diseases require radical new funding methods article 2018/11/18
Rare diseases prompted care in ancient times article 2019/07/28
Rare diseases more common than we think article 2019/11/10
Rare diseases in China, their high treatment costs, and the people publicising patients’ plight article 2019/04/12
Rare Diseases in Asia-Pacific article 2020/08/02
Rare diseases are not uncommon, and may be the mother lode for the world’s pharmaceutical producers and researchers article 2019/08/05
Rare diseases are not uncommon, and may be the mother lode for the world's pharmaceutical producers and researchers article 2019/07/09
Rare Disease Trials Require Patient Insights And Input article 2018/10/16
Rare Disease Therapy Development and Access Remain Top FDA Priorities During COVID-19 article 2020/06/11
Rare Disease R&D Investments Likely To Grow In Biopharma article 2019/05/02
Rare Disease R&D Investments Likely To Grow In Biopharma article 2019/05/08
Rare Disease Q&A: What Rare Diseases Are and Why That Matters article 2019/12/15
Rare disease drove two women on a mission to change FDA drug review process article 2020/07/19
Rare Disease Doctors Are Struggling. Here's 3 Ways Patients and Families Can Help. article 2020/05/24
Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest article 2020/08/08
Rare disease acromegaly finds an Ottawa champion article 2018/11/18
Randall Rutta: Discrimination against people with rare diseases article 2020/01/26
ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa article 2019/12/12
ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP) article 2019/08/17
Priscilla Chan talks her new 'Rare As One' initiative to combat rare diseases video 2019/09/09
Potential treatment for rare degenerative disease discovered article 2020/08/02
Potential Gene Therapy Safely Restores FVIII Clotting Factor in Animals and Human Cells, ASC Therapeutics Reports article 2019/05/08
PODCAST: Rare Unplugged: Episode 1 podcast 2018/07/30
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
Podcast Extra: The quest for a rare disease treatment podcast 2019/01/15
Pfizer Steps Further In The Door For Gene Therapies article 2019/03/25
Patients: The Catalysts Behind Rare Disease Research & Drug Development article 2018/10/28
Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial article 2018/10/16
PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights article 2019/05/08
Opinion: Value-based agreements could disrupt how we pay for new therapies article 2019/04/12
One in 67 Hongkongers suffers from a rare disease, study finds article 2018/09/25
One in 67 Hongkongers suffers from a rare disease, study finds article 2018/10/03
Once free, drug for rare disease now being sold for $375K a year video 2019/02/11
North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte article 2018/07/24
NORD Launches Financial Assistance Program for Rare Disease Community Members Impacted by COVID-19 article 2020/04/25
NIH-supported research survey to examine impact of COVID-19 on rare diseases community article 2020/05/10
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research article 2019/03/06
NIH funding bolsters rare diseases research collaborations article 2019/10/05
Niemann-Pick Disease: Reno twins with rare, fatal disease will be on CNN show "Something's Killing Me" article 2018/08/17
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases article 2018/09/10
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases article 2018/09/10
New Study Proves the Internet Can Help You Get a Rare Disease Diagnosis article 2019/08/20
New Hope for Rare Mitochondrial Diseases: An Interview With Minovia Therapeutics’ Natalie Yivgi Ohana article 2019/07/23
Navigating the Rare Disease Space: Insights from a Danish CEO article 2018/12/19
Nature Outlook: RNA Therapies article 2020/02/02
Nature Outlook: Huntington's Disease article 2018/10/28
Nature Outlook: Huntington's Disease article 2018/10/28
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
My life with face blindness article 2019/09/01
My friend chose an assisted death in Switzerland. Her dying wish was to tell you why article 2020/06/21
My Constant Insomnia Turned Out to Be a Symptom of a Rare Disorder Called Hypophosphatasia article 2020/07/29
MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte article 2018/07/18
Moyamoya: The rare disease you've never heard of video 2019/01/20
Most ER Staff Don't Understand Rare Disease, So This Is How I Advocate for Myself article 2019/11/24
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist article 2019/03/18
Montreal doctors’ 'Hail Mary’ treatment could save babies' lives from most common of all rare diseases article 2019/05/08
Mom Is Tirelessly Raising Awareness About Her Daughter's Rare, Life-Threatening Disease article 2019/04/20
Mom Blazes Path to Cure for Son’s Rare Disease article 2019/03/06
Mitochondrial Disease: 13 Year Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/09/25
Mindfulness training shows promise for people with MS article 2020/05/26
Mercer Island family raises awareness for rare, undiagnosed diseases article 2020/02/23
Meet the man who invented Viagra: He's fighting rare diseases with artificial intelligence to treat patients video 2020/02/09
Meet the family working with scientists to fight rare diseases with CRISPR therapy video 2020/06/21
Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research article 2018/12/09
Medicine by machine: Is A.I. the cure for the world’s ailing drug industry? article 2020/02/09
Medical foods are vital to treat rare diseases — but some insurers won't pay article 2018/09/25
Medical Detectives: The Last Hope For Families Coping With Rare Diseases article 2019/01/05
Medical Detectives: The Last Hope for Families Coping with Rare Diseases article 2018/12/19
Med student uses 'Zebra Alphabet' to bring awareness to rare diseases article 2018/09/03
Man diagnosed with rare disease takes 2 life-changing steps article 2018/12/09
Lysosomal Acid Lipase Deficiency Financial Assistance Program Launched by The Assistance Fund article 2019/01/26
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
Local Hero with rare disease to be honored at 2018 MG Walk at Bear Creek Pioneers Park article 2018/11/18
Local family shares their journey battling rare disease article 2018/10/19
Live bacteria deliver crucial enzymes straight to the gut article 2018/12/22
Life for a child with a rare disease is a grueling roller coaster ride: A mother explains article 2019/03/06
Lewisville family helps to raise awareness for rare disease article 2020/07/01
Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease article 2018/08/15
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
Lack of treatment options for rare diseases prompts call for change article 2018/09/10
L1 Cam Syndrome: $1K Needed To Help Dublin Boy With Rare Disease In Need Of Bike article 2018/08/20
Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk article 2018/08/14
Internet can be valuable tool for people with undiagnosed rare disorders article 2019/08/11
Inspirational boy battling rare disease returns from hospital after 'life-saving' surgery article 2020/04/19
Infographic: Which Rare Diseases Are The Most Common? article 2019/09/22
In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical Relief Program article 2020/05/17
In developing new drugs for rare diseases, this non-profit steps in when pharmaceutical companies won't article 2019/01/15
In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder podcast 2019/07/30
Immune Thrombocytopenic Purpura in a Patient with Covid-19 article 2020/04/30
ICER discriminates against people with rare diseases article 2019/10/05
I want to cure my son of his rare genetic disease. Is that wrong? article 2020/06/21
I have a rare disease. This is my hope for the future of medicine article 2019/05/19
I have a rare disease. Research on such illnesses can provide insights into more common ones. article 2019/02/19
Hurler Syndrome: World record attempt aims to help local boy battle rare disease article 2018/08/21
Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report article 2018/07/10
Hunter Syndrome: Talking about rare disease article 2018/08/13
Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana article 2018/07/17
How UK startup Mendelian hopes to use machine learning to diagnose rare diseases article 2019/04/20
How People With Weakened Immune Systems Are Navigating The Coronavirus podcast 2020/03/21
How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025 article 2018/11/18
How FDA's Breakthrough Therapy Designation Program Changed the Rare Disease Space article 2018/10/28
How coronavirus affects young adults with chronic illnesses and rare diseases article 2020/07/26
How Can We Improve the Diagnosis and Treatment of Rare Diseases? article 2020/03/15
How artificial intelligence can help detect rare diseases article 2019/06/16
How a Collaborative Ecosystem is Driving the Development of Successful Therapies in Rare Disease Research article 2018/09/11
Hermansky-Pudlak Syndrome: MN man battles rare genetic disorder article 2018/07/31
Hereditary Angioedema: Shire wins FDA approval for potential blockbuster rare disease drug article 2018/08/24
Helping Patients With Rare Dieases During the Coronavirus Pandemic video 2020/04/12
Hearing loss and rare diseases: What's the connection? article 2019/05/19
Healx Pushing New Drug Development Model in Rare Diseases with Barth Syndrome Foundation article 2019/03/01
He's the oldest-known man with rare Proteus syndrome and he hopes to help find a cure article 2020/02/02
Have 12 friends? You probably know someone with a rare disease article 2019/08/11
hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease article 2018/08/10
Guillain–Barré Syndrome Associated with SARS-CoV-2 article 2020/04/25
Guillain-Barre Syndrome: What we know about the disease Travis Frederick is fighting article 2018/08/25
Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland article 2018/07/12
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19 article 2020/03/21
Genomics in healthcare: How systems are using the data and whether there is a cause for concern article 2020/03/12
Genetic screening for newborns yields some answers, more questions article 2019/01/05
Gene therapy to halt rare form of sight loss article 2020/02/17
Gene therapy targets sickle-cell disease article 2019/04/04
Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports article 2020/06/10
GBT Snags FDA Approval for First-of-its-Kind Sickle Cell Disease Treatment article 2019/11/27
Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech article 2018/08/19
Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org article 2018/03/07
First US attempt to cure a rare disease with genome editing fails miserably article 2019/02/10
First Signs of Huntington's Disease Detectable Via a New Blood Test article 2018/10/17
Finding New Uses For Old Drugs - A Lifeline For Rare Disease Patients article 2019/03/01
Finding Answers: Statesville woman's rare disease needs rare treatment article 2019/01/05
FDA's Banner Year For Approvals: Will Market Access To New Drugs Follow Suit? article 2019/01/26
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases article 2019/02/02
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 is Working to Bridge Gaps and Meet Needs for Rare Disease Product Development article 2019/03/18
FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development article 2020/01/18
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 Awarding Grants for Research into Rare Diseases article 2018/09/25
FDA Awarding Grants for Research into Rare Diseases article 2018/09/26
FDA approves treatment for patients with rare bone marrow disorder article 2019/08/17
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
FDA approves pexidartinib for tenosynovial giant cell tumor article 2019/08/02
FDA Approves Orphan Drug Pemigatinib for Rare Bile Duct Cancer Cholangiocarcinoma article 2020/06/10
FDA Approves Oral Treatment for Spinal Muscular Atrophy article 2020/08/07
FDA approves new treatment for rare disease of the bone marrow and blood article 2019/01/05
FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord article 2020/06/14
FDA Approves New Therapy for Myelodysplastic Syndromes (MDS) That Can Be Taken at Home article 2020/07/07
FDA Approves New Therapy for Dravet Syndrome article 2020/06/25
FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease article 2020/08/04
FDA approves first treatment to increase pain-free light exposure in patients with a rare disorder article 2019/10/13
FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease article 2018/11/28
FDA approves first treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system article 2019/06/30
FDA approves first treatment for inherited rare disease article 2019/12/12
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels article 2019/09/29
FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder article 2019/05/12
FDA Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease article 2020/06/16
FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease article 2020/04/12
FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL article 2020/07/29
FDA Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC article 2018/07/29
Fanconi Anemia: Jimbo Fisher spreads awareness of son's blood disease through nonprofit article 2018/08/31
Familial Chylomicronemia Syndrome: Ultra-rare disease patients find support in connectedness article 2018/08/21
Familial Amyloid Polyneuropathy: What caused this man's scalloped pupil? article 2018/07/31
Fabry Disease: FDA Approves Galafold for Rare Genetic Disorder, Fabry Disease article 2018/08/14
Experimental Therapy Gives Children With Genetic Disorders A New Chance At Life video 2019/03/25
Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/10/05
Exclusions: Adding Complexity for Patients with Rare Diseases article 2018/10/10
Exclusions: Adding Complexity for Patients With Rare Diseases article 2018/12/25
Evans syndrome: Everything you need to know article 2020/02/23
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients article 2019/06/22
European initiative targets diagnosis, treatment of rare diseases article 2019/08/05
European Commission approves nintedanib for the treatment of systemic sclerosis-associated interstitial lung disease (SSc-ILD) article 2020/04/25
EU support for Italian biotech's red blood cell technology to treat rare diseases article 2020/08/08
Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun article 2018/07/29
Epidermolysis Bullosa: 'My skin tears and blisters at the slightest touch, but it won't stop me from modelling' article 2018/08/30
Epidermodysplasia Verruciformis: New Link Found Between A Rare Skin Disease and Beta-HPV article 2018/08/16
Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies article 2020/03/29
Ehlers-Danlos Syndrome: Madera boy with rare disease using YouTube to raise awareness article 2018/09/06
East Bay family launches national campaign to save life of 5-year-old who needs bone marrow transplant article 2019/01/20
Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat article 2018/07/19
Dublin teen's film on rare disease chosen for international festival article 2020/04/12
Dublin Boy With Rare Disease Gets Special Bike article 2018/11/28
Dubai Science Park Raises Focus on Rare Diseases During Expert Panel article 2018/09/30
Drug shortage threatens health of O'Fallon man video 2019/07/09
Drug offers hope for patients with primary hyperoxaluria type 1 article 2020/06/08
Drug Discovery for Rare Diseases article 2019/06/22
Drew Brees gives emotional speech as Steve Gleason receives Congressional Gold Medal article 2020/01/18
Dravet Syndrome: Community rallies around 3-year old battling rare disorder article 2018/08/25
Dr. Google' Helps Some Patients Diagnose a Rare Disease article 2019/09/01
Doctors said boy wouldn't live past his second birthday. His town just threw a parade for his third article 2019/11/24
Developing RNA Medicines for Rare Genetic Diseases article 2018/09/11
Designer Microbes: a Potential Living Cure for Crohn's and Phenylketonuria article 2019/04/21
Denver family fundraising to help toddler suffering from rare disease video 2019/08/05
Denali's Hunter Syndrome Candidate Gets Orphan Drug Status article 2019/06/16
Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan article 2018/07/24
Dansville teen overcomes rare disease, returns to dance article 2018/12/09
CZI awards $1.3 million to support the global rare disease community article 2020/07/19
Cystinosis: Firelight Fund to help local family as child battles rare disease article 2018/09/11
COVID-19 pandemic hinders access to care for patients with rare diseases article 2020/05/10
COVID-19 in a patient with systemic sclerosis treated with tocilizumab for SSc-ILD article 2020/04/02
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say article 2020/05/30
Could This Radical New Approach to Alzheimer’s Lead to a Breakthrough? article 2019/02/10
Could gene therapy cure sickle cell anemia? video 2019/03/11
Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com podcast 2018/07/19
Commercialization Imperatives for Success in Rare Diseases article 2019/09/15
Commentary: What those with rare diseases want us to consider article 2020/05/05
Coalition Will Address Racial Disparities in Rare Disease Communities article 2020/06/14
CMN: 'For someone to see the way I look as art is amazing' article 2019/03/18
China is picking up the fight against rare diseases article 2019/06/30
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
Chiesi creates Boston rare disease unit ahead of Fabry launch article 2020/02/09
Cheers, tears as Staten Island teen with rare disease receives new home article 2018/11/28
Change the genes to fix the skin article 2019/04/04
Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases article 2020/02/09
Chan Zuckerberg Initiative launches new program to support rare disease patients article 2019/06/16
CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com article 2018/07/12
California parents hope for miracle after baby diagnosed with rare, fatal genetic disorder article 2019/05/19
California group helps people with rare diseases aim for new heights article 2018/08/16
CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo article 2018/07/16
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
Boy's Severe Headaches Lead to Rare Eye Disease Diagnosis article 2019/09/01
Boehringer Ingelheim Gambles $2.62 Billion on Two Fibrotic Disease Deals article 2019/07/23
Biallelic HMBS Deficiency: 'Kure 4 Kam': Parents navigate medical maze so 1-year-old son might withstand rare disease article 2018/08/24
Benefit held for local woman battling rare disease video 2018/11/18
Batten Disease: Norfolk Tides help Virginia Beach teen with rare disease article 2018/08/21
Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes article 2016/06/27
Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes article 2016/07/07
Axovant Licenses Two Gene Therapies Aimed at Tay-Sachs and Sandhoff Disease article 2018/12/19
Autosomal Dominant Alzheimer's Disease: Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/09/25
Army major walking barefoot from Cornwall to Edinburgh to fund treatment for daughter with rare disease article 2020/07/11
Apple to Participate in Meeting Advocating for Better Patient Access to Health Info article 2020/01/26
Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant article 2018/08/17
Apic Bio’s APB-102 Receives Orphan Drug Designation from the FDA for the Treatment of Genetic SOD1 ALS article 2019/07/23
Antioch teen with rare disease won’t let COVID-19 stop her article 2020/03/21
Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia article 2018/07/21
Angelman Advocate Launches ‘Combined Brain’ Rare Disease Consortium article 2019/12/12
An experimental test may help confirm cases of chronic fatigue syndrome article 2019/05/02
An experimental test may help confirm cases of chronic fatigue syndrome article 2019/05/08
Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington article 2018/07/29
Alnylam's Givosiran Looks Positive Overall in Phase III Trial for Rare Disease article 2019/03/07
Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria article 2018/08/01
Alexion and Affibody Announce Partnership to Co-Develop Anti-FcRn Affibody® Molecule article 2019/03/25
AI technology can identify genetic diseases by looking at your face, study says article 2019/01/15
AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu article 2018/07/26
Advances in inborn errors of metabolism article 2019/01/26
Adult Polyglucosan Body Disease: Film series pick highlights rare disease - Jewish Exponent article 2018/07/25
Addressing Diagnosis Challenges in the Rare Disease Space article 2018/10/19
Acute Necrotizing Encephalopathy: Moree community supports two-year-old Paige Thomas as she fights rare brain disease article 2018/08/21
Access And Actionability Are Key For Genetic Testing And Precision Medicine article 2020/01/18
A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment podcast 2019/12/28
A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving podcast 2020/06/27
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
A rare-disease entrepreneur follows an unconventional drug development path article 2018/10/19
A Rare Human Disease Has Been Found in Dinosaur Bones, Could it Lead to a Treatment? article 2020/02/19
A Helpful Online Safety Guide for People With Autism Spectrum Disorders article 2020/07/01
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
A Genetic Test For A Microscopic Problem Came With A Jumbo Price Tag article 2020/04/05
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived article 2020/08/02
7 rare diseases a rural Wisconsin doctor found in his Amish and Mennonite patients article 2019/10/19
7 Pro Tips to Help Deal With the Anxiety of Rare Disease Parenting article 2019/12/28
5 Things You Should Know About Vasculitis article 2020/03/21
5 Things You Should Know About Stiff-Person Syndrome article 2020/05/30
5 Questions: Stephen Montgomery on RNA’s role in diagnosing rare diseases article 2019/06/22
4 deeply personal stories about the impact of genetic testing article 2020/01/26
27 Years of Humanitarian Programs in Vietnam article 2019/12/22
25-year-old faces rare form of ALS that took her twin, but experimental drug offers hope article 2019/06/22
15-month-old Denver girl fighting rare disease, raising awareness for treatments and a cure article 2019/01/05
13-Year-Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/10/04
13 Things ER Staff Should Know About People With Rare Diseases article 2019/08/25
'Revolutionary' DNA test can detect thousands of rare diseases in children article 2020/01/03
'It Will Consume Your Life': 4 Families Take On Rare Diseases article 2020/07/11
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington article 2018/10/03