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Title Category News Date Link
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