Title Category Date Link
Persistence leads to diagnosis of ultra-rare disease Article May 10, 2024
In Washington DC and Gaza two very different families are united by one very rare disease Article May 10, 2024
Rare disorder causes man to see people's faces as 'demonic' Article May 10, 2024
Jaguar Health Announces Submission of Clinical Trial Applications for Crofelemer for the Rare Disease Indications Microvillus Inclusion Disease (MVID) and Short Bowel Syndrome (SBS) in Europe Article May 10, 2024
These congressmen want to modify the Inflation Reduction Act to foster rare disease research Article May 10, 2024
Imagine living in a 4-foot body that doesn't develop chronic diseases Article May 3, 2024
To save lives, the FDA must change how it evaluates rare disease treatments Article May 3, 2024
AI Might Spot Rare Diseases in Patients Years Earlier Article May 3, 2024
Pilot program improves well-being of families during advanced care planning Article May 3, 2024
AI Is Transforming Drug Matching for Cancer, Rare Diseases — Here's How Article April 27, 2024
As more rare disease therapies launch, their prices are rising Article April 19, 2024
Peter Frampton rocks on despite potentially crippling disease: ‘I’m a fighter,’ says the 74-year-old guitar great Article April 12, 2024
Woman with ‘one-of-a-kind’ condition can finally see her own face again after more than 60 hours of surgery Article April 6, 2024
Gene Therapy for Rare Neurodegenerative Disease Shows Promise in Trial Article March 21, 2024
A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access Article March 21, 2024
When Should a Pediatrician Suspect a Rare Disease? Article March 14, 2024
New way for states to cover pricey gene therapies will start with sickle cell disease Article March 14, 2024
Big league bartenders a hit at annual fundraiser for rare disease Article March 14, 2024
FDA approves new treatment for rare liver disorder Article March 14, 2024
Communicating About Rare Diseases Article March 9, 2024
My daughter has a rare disease. We shouldn't have had to leave the US to save her life. Article March 9, 2024
Many parents of kids with rare diseases fight for research — even if it’s too late for their child Article March 1, 2024
We Speak Duchenne Video March 1, 2024
What It’s Like to Live With a Rare Liver Disease Article February 25, 2024
Guest Commentary: Rare diseases affect 30 million Americans. We must answer the challenges to finding cures Article February 25, 2024
‘Empowering': Rare Disease Week on Capitol Hill to draw calls for change Article February 25, 2024
Amy Schumer has been diagnosed with Cushing syndrome. What to know about the rare disorder Article February 25, 2024
Hailey boy discusses his bout with a rare disease Video February 17, 2024
Opinion: Rare diseases affect 30 million Americans. These are the challenges to find their cure. Article February 11, 2024
What is Morgellons disease, the mysterious condition Joni Mitchell claims she has? Article February 4, 2024
New CRISPR center brings hope for rare and deadly genetic diseases Article January 27, 2024
FDA’s Marks Advocates for Flexibility in Rare Disease Gene Therapy Trials Article January 27, 2024
Rare Disease Patient Photo Contest Article January 27, 2024
Kernersville woman battling rare disease enrolled in clinical trial Article January 27, 2024
Ohio mother hopes for a cure to save her son, 8, from rare, fatal disease: ‘Gut-wrenching’ Article January 27, 2024
What to know about acquired von Willebrand disease (AvWD) Article January 27, 2024
Boston Children's Hospital lab trying to develop drug to treat boy's deadly rare disease Video January 20, 2024
United States Food & Drug Administration (FDA) Grants Mesoblast Rare Pediatric Disease Designation for Revascor® (Rexlemestrocel-L) in Children With Congenital Heart Disease Article January 20, 2024
mRNA Technology Emerging for Rare Diseases Article January 17, 2024
The Million Dollar Bike Ride for Rare Diseases Article January 17, 2024
FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia Article January 17, 2024
Girl with rare disease 'thriving' after therapy Article January 17, 2024
A Doctor And Mother's Plea To FDA To Help Save Children With Rare Disease Article January 7, 2024
Scientists Discover Rare Mutation That Halves Risk of Parkinson's Disease Article January 7, 2024
What Is Myelofibrosis? Article December 31, 2023
Gene Delivery in Canavan Disease: Innovative Leaps in Technology Article December 31, 2023
Alexander disease: Causes, symptoms, and diagnosis Article December 23, 2023
Our Impact on Rare Diseases Article December 17, 2023
FDA approves gene therapy for sickle cell disease: 'One incredible journey of success.' Article December 12, 2023
7-year-old Bucks County girl with rare disease enjoys holidays for first time thanks to new medication Article December 3, 2023
Beyond the Diagnosis: Putting a face to children with rare diseases Article December 3, 2023
Soligenix Receives FDA IND Clearance for Phase 2 Clinical Trial of Dusquetide in the Treatment of Aphthous Ulcers in Behçet's Disease Article December 3, 2023
Babies with rare disease to survive past first birthday with new drug available on NHS Article December 3, 2023
Study Highlights Poor Health Outcomes in Older Adults with Hemophilia Article November 25, 2023
Pilot launched to support children with rare conditions to access personalised therapies Article November 25, 2023
Researchers find new therapy that can help treat rare, hereditary diseases Article November 25, 2023
The rare disease films raising greater awareness Article November 18, 2023
FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder Article November 11, 2023
Overcoming the ongoing challenges for rare disease patients in the UK Article November 11, 2023
Rare diseases: Visibility through art Article November 11, 2023
How Common Is Gaucher Disease? Article November 11, 2023
His Rare Disease's Cure Was Sitting on the Pharmacy Shelf Article November 4, 2023
The North America Rare Disease Summit focused on the need for centralized data to improve the lives of 30 million people living with rare conditions Article November 4, 2023
Fairport woman with rare disease searches for living liver donor Article November 4, 2023
IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases Article October 27, 2023
A bright future ahead for rare kidney diseases Article October 27, 2023
On a journey to diverse, inclusive, and more efficient rare disease trials Article October 27, 2023
Reps. Kelly, Matsui, Dunn, Thompson introduce legislation to support access to evidence-based care for rare disease patients Article October 27, 2023
Complications You May Experience If You Have Crohn's Disease Article October 27, 2023
‘We had no hope’: Patients, advocates testify at U.S. Senate hearing in support of changing FDA rules for rare disease treatments Article October 27, 2023
Global Genes and the Rare Disease Diversity Coalition Expand Effort to Accelerate Diagnosis of Rare Disease in Underserved Communities Article October 21, 2023
Former attorney works to bring resources to those with rare genetic disease Article October 21, 2023
National Organization for Rare Disorders Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development Article October 21, 2023
Lexi Reed Says She's 'Thankful' for Every Scar: 'I See Battle Wounds' Article October 15, 2023
Baltimore woman defies science, runs marathons with rare autoimmune disease Video October 15, 2023
National Organization for Rare Disorders Host 2023 Breakthrough Summit Article October 15, 2023
Life-changing surgery: Doctor disconnects brain of 6-year-old with rare disease Article October 15, 2023
Novartis says positive interim results on rare kidney disease drug Article October 9, 2023
Creating an iPS cell resource for rare and intractable diseases Article October 9, 2023
American Kidney Fund Launches Educational Awareness Campaigns for Two Rare Kidney Diseases Article October 1, 2023
VMware: Finding Treatments for Children With Rare Diseases Article October 1, 2023
Travis Barker says he suffered a facial pain syndrome: What is trigeminal neuralgia? Article October 1, 2023
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies Article October 1, 2023
Canadian Organization for Rare Disorders supports call on United Nations member states to turn universal health coverage into a reality for people living with rare diseases Article September 23, 2023
Nearly one in ten Americans will be hit with a “rare” disease Article September 23, 2023
Kathleen Folbigg: Misogyny helped jail her, science freed her Article September 23, 2023
Democrat Jennifer Wexton Announces Retirement Following Rare Diagnosis Article September 19, 2023
Small molecule drug shows promise in rare disease Article September 16, 2023
Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient Article September 16, 2023
Mark Zuckerberg and Dr. Priscilla Chan’s plan to prevent, cure, and manage all diseases by 2100 Article September 16, 2023
'Collectively, we make a big community': 2 Portage residents serve on rare disease council Article September 16, 2023
How one woman's rare disease experience inspired her career Article September 16, 2023
How whole genome testing saved the life of a baby with a rare disease, and why such tests could help millions more with rare genetic disorders Article September 3, 2023
UC San Diego Launches Gene Therapy Initiative, Targeting Treatments for Rare Diseases Article September 3, 2023
The Transformative, Alarming Power of Gene Editing Article September 3, 2023
Rare Diseases Podcast August 26, 2023
A move to cut drug prices has patients with rare diseases worried Article August 26, 2023
What Is Huntington Disease? Article August 26, 2023
A broad genetic test saved one newborn’s life and research suggests it could help millions of others Article August 26, 2023
Scientists genetically decode rare kidney disease Article August 26, 2023
Rare disease patients report great challenges in many areas of life Article August 19, 2023
4 years later, Ipsen's 'de-risked' rare disease drug Sohonos finally gains FDA approval Article August 19, 2023
US FDA approves Regeneron's ultra-rare blood disease drug Article August 19, 2023
Pennsylvania mother and son both born with rare genetic disease: 'Closer because of this' Article August 12, 2023
Liver donation brings hope, friendship to 2 Prince William Co. women Article August 12, 2023
3 of 4 children in a Molalla family have rare progressive disorder Video August 12, 2023
Opinion: To many treatable diseases go unnoticed. This could change that. Article August 6, 2023
Chinese families being 'destroyed' by burden of facing rare disease ALS Article August 6, 2023
Not So Rare After All: Unveiling the True Prevalence of “Rare” Diseases Article August 6, 2023
Leprosy Outbreak in Florida: What You Need to Know Article August 6, 2023
Asian countries prioritise rare disease market access amid stark disparities Article July 22, 2023
High-tech clinic in rural Amish community helps children with rare genetic disorders Video July 22, 2023
Teen uses social media to document rare disorder that causes face to waste away Video July 22, 2023
Life saved: AI discovers existing drug works for rare disease Article July 15, 2023
Whole Genome Sequencing Boosts Diagnosis of Rare Disease in Infants Article July 15, 2023
Solving rare disease mysteries ... and protecting privacy Article July 15, 2023
Woman With Ultrarare Rare Disease Gives Birth Through IVF in 'World First' Article July 15, 2023
Early Success: mRNA & CAR T Therapy To Treat Rare Autoimmune Disease Myasthenia Gravis Article July 9, 2023
Uncommon but Affecting Millions: The Rare Disease Paradox Article July 4, 2023
PTC Therapeutics says interim data for Huntington's disease drug shows promise Article June 24, 2023
FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy Article June 24, 2023
What is ‘Viking disease’? Deforming hand disorder linked to Neanderthals Article June 18, 2023
I suffer from the world's most beautiful disease - and also the most expensive to treat Article June 18, 2023
Genomics Are a Lifesaver for Patients With Rare Diseases Article June 11, 2023
SoCal toddler with rare genetic disorder inspires resiliency, research and hope Video June 11, 2023
Gunnar Esiason's story highlights importance of clinical trials | Opinion Article June 11, 2023
A new research effort takes aim at 8 rare diseases. It could revolutionize many more. Article May 27, 2023
Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars Article May 27, 2023
What is stiff person syndrome, the condition Celine Dion is battling? Article May 27, 2023
Rare Disease: Could Existing Drugs Turn the Tide? Article May 21, 2023
FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa Article May 21, 2023
Ohio boy, 7, battles rare disease, writes book Article May 21, 2023
Widow sheds light on CJD after rare disease takes Michigan man’s life Video May 21, 2023
A new, more diverse human genome offers hope for rare genetic diseases Article May 14, 2023
FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease Video May 14, 2023
Mom donates kidney to pediatrician daughter suffering from rare disease Video May 14, 2023
Michael J. Fox Reveals Private Journey with Parkinson's Disease in Trailer for 'Still' Documentary Video May 6, 2023
Norwich teens manage rare disease phenylketonuria Video April 30, 2023
Gene therapy offers hope for rare diseases. But the stories don't always have happy endings. Video April 30, 2023
Fighting rare diseases: New Hartford boy’s battle with Ohtahara syndrome Video April 30, 2023
Drug for rare form of Lou Gehrig's disease OK'd by FDA Article April 30, 2023
Girl to get life-saving treatment for rare immune disease Video April 30, 2023
Erasing or replacing errors in a patient’s genetic code can treat and cure some genetic diseases Article April 22, 2023
How AI Could Make Every Disease A Rare Disease Article April 22, 2023
5,500 people diagnosed with rare genetic disorders in major UK and Ireland study Article April 15, 2023
Family of Naperville girl with Wolf-Hirschhorn Syndrome aims to raise awareness of rare disease Video April 15, 2023
Improving Health Equity For Rare Diseases Article April 8, 2023
6-Year-Old Enjoys Fundraiser for Her Rare Disease: I Wore 'a Unicorn Onesie and Butterfly Wings!' Article April 8, 2023
“A Second Chance at Life”: Can Gene Therapies Beat Rare Disease? Article April 1, 2023
How Rare Disease Patients Are Fighting Their Conditions Head-On Article April 1, 2023
England's NICE signs off on PTC Therapeutics' $3.7M gene therapy for ultra-rare disease Article March 25, 2023
Rare-disease patients battle common issues Article March 25, 2023
U.S. FDA approves Sanofi's bleeding disorder therapy Article March 25, 2023
Genetic causes of three previously unexplained rare diseases identified Article March 18, 2023
I Lost My Daughter To A Rare Disease, But It's What I Did After That Surprises People The Most Article March 12, 2023
Rare disease rap: Patient advocate writes song to raise awareness Video March 12, 2023
I went from squatting 350 pounds to completely paralyzed at 26 in a matter of weeks. Doctors told me it was a herniated disk, but it was Guillain-Barré. Article March 12, 2023
Acadia Awaits Potential Approval of First Rett Syndrome Medicine Article March 12, 2023
Heartbreak of children lost to rare disease for which there was no cure Article March 4, 2023
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.? Article March 4, 2023
A scientist’s daughter was born with a rare disease, so he began to study it. An anonymous donor just chipped in $25 million Article March 4, 2023
FDA Approves First Therapy for Friedreich's Ataxia (Updated) Article March 4, 2023
Explainer: What Is Europe's Rare Disease Moonshot? Article February 26, 2023
The Reality of Living with a Rare Disease: Emily’s Ongoing Battle Article February 26, 2023
Mental health: Mum with rare disease says support is lacking Article February 26, 2023
Who can afford that'? Patients face costly bills amid FDA's battle over 'orphan drugs' Article February 20, 2023
Girl with rare deadly disease receives revolutionary million-dollar gene-therapy drug – but it is too late for her older sister Article February 20, 2023
2022 was a breakthrough year for understanding rare diseases. 2023 needs to be better Article February 12, 2023
Rare genetic disease may protect Ashkenazi Jews against TB Article February 12, 2023
A Rare Neurological Disease Involving Cellular Recycling Discovered Article February 12, 2023
A 28-year-old woman thought an old shoulder injury was flaring up. She was actually having mini-strokes due to a rare brain condition. Article February 12, 2023
Gene therapy gel heals decades-old wounds in trial for blistering skin disease Article February 4, 2023
Huntington's Program Bites the Dust as Novartis Cleans House Article February 4, 2023
Bright Ideas for Rare Disease Day 2023 Article February 4, 2023
Gene therapy gel heals decades-old wounds in trial for blistering skin disease Article January 29, 2023
Drug repurposing emerges as viable option for rare disease treatment Article January 29, 2023
Rare Infectious Diseases: A Tutorial Article January 29, 2023
Gene therapy into brain helps kid with rare disease Video January 29, 2023
What Causes Parkinson's Disease? Article January 29, 2023
Women are finding out their 'rare' health issues aren't actually that uncommon thanks to social media Article January 14, 2023
High school sweethearts tackle rare genetic disease Video January 14, 2023
Girl with rare disease beats the odds to celebrate 5th birthday Article January 14, 2023
Aspen’s story: A 4-year-old living with a rare disease Article January 7, 2023
Model, 24, with Rare Skin Disease Bares Scars to Raise Awareness: 'I Choose to Show the Body I Was Given' Article January 7, 2023
Rare diseases and space health: optimizing synergies from scientific questions to care Article December 30, 2022
Families Push Research Forward in Rare Diseases Article December 30, 2022
The Rare Disease Rallying Cry: If Not Me, Who? Article December 30, 2022
Concord woman with rare disease who faced deportation allowed to stay in U.S. permanently Article December 30, 2022
Oprah Winfrey's Hashimoto's Disease Diagnosis Explained Article December 30, 2022
Jameela Jamil discusses experience with Ehlers-Danlos syndrome on TikTok Article December 30, 2022
Millions have the same ‘bendy body’ disease as my daughter. Why isn’t the medical profession paying more attention? Article December 30, 2022
Rare Parents Tackling Rare Diseases Article December 24, 2022
They Created a Drug for Susannah. What About Millions of Other Patients? Article December 24, 2022
The Reasons Why I Share Our Rare Disease Story Article December 24, 2022
In a first, children with rare genetic diseases get mitochondrial transplants from their mothers Article December 24, 2022
Man Paralyzed from the Neck Down from Rare Disease Makes Incredible Recovery, Now Back at the Gym Article December 24, 2022
Sequencing projects will screen 200,000 newborns for disease Article December 17, 2022
The Rare World of Rare Diseases Article December 17, 2022
Rare Disease Finding Sheds Light On Head And Neck Cancer Article December 10, 2022
Celine Dion reveals rare neurological disease in emotional video message Article December 10, 2022
Scientists Finally Discover the Cause of a Rare Brain Disease Article December 3, 2022
With help from Duke doctors, a rare genetic disease is treated in the womb for the first time Article December 3, 2022
A 27-year-old with a rare disease has died in an experimental gene-editing study. He was the only volunteer. Article December 3, 2022
Varun Dhawan To Salman Khan: 5 Bollywood Celebs Who Battled Rare Diseases & Came Out Victorious Article December 3, 2022
Lucy has a rare genetic disorder. Two doctors are leading the desperate hunt for a cure: her parents Article December 3, 2022
Researchers Track an Ultra-Rare Disease That Turns Muscle to Bone Article December 3, 2022
Mental Health and Rare Diseases Article December 3, 2022
Graves' Disease Explained: Causes Symptoms, And Treatment Article December 3, 2022
What to know about Krabbe disease Article December 3, 2022
How AI is finally helping rare diseases gain more than just attention Article December 3, 2022
Missing pathway in lysosome underlies newly discovered human disease Article December 3, 2022
New uses for old drugs? Every Cure offers hope for people with rare diseases Article September 24, 2022
For Some ALS Patients, A New Drug Shows Potential To Slow And Even Reverse Disease Progression Article September 24, 2022
Tiny but mighty' Stamford child with rare joint disease comes home after 5 years in long-term care Article September 24, 2022
U.S. FDA approves bluebird bio's gene therapy for a rare neurological disorder Article September 17, 2022
Scientists discover novel mechanism that causes rare brain disease Article September 17, 2022
FDA, NIH Launch Public-private Partnership For Rare Neurodegenerative Diseases Article September 17, 2022
What to Know About Capillary Leak Syndrome Article September 10, 2022
New Parkinson’s test developed thanks to woman who could smell the disease Article September 10, 2022
Rare Disease Report Podcast: CDKL5 Deficiency Disorder Podcast September 2, 2022
Sanofi, after long research journey, wins FDA approval for rare disease drug Article September 2, 2022
San Diego woman with rare disease receives first breakthrough treatment at UCSD Video August 28, 2022
The Most Expensive Drug in the US is a Rare-Disease Game Changer Article August 28, 2022
Drug Development for Rare Diseases is Littered with Regulatory Roadblocks Article August 21, 2022
bluebird bio Announces FDA Approval of ZYNTEGLO®, the First Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions Article August 21, 2022
What is Rett syndrome? Richard Engel mourns death of 6-year-old son Henry Article August 21, 2022
Cure Rare Disease Scores IND for First-in-Human CRISPR Therapeutic Article August 14, 2022
‘Abnormal’ Protein Could Be Common Link Between All Forms of Motor Neuron Disease Article August 14, 2022
Couple speaks out on decision to get abortion after fetus diagnosed with rare genetic conditions Video August 10, 2022
Poor health-related quality of life can lead to rare diseases Article August 7, 2022
Gaining insights into spastic paraplegia Article August 7, 2022
Designing a tool for better diagnosis of rare and genetic diseases Article August 7, 2022
Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne Muscular Dystrophy Article August 7, 2022
Charlotte family launches foundation to raise awareness of rare disease Video August 7, 2022
What You Need to Know About This Rare Chronic Autoimmune Disease Article August 7, 2022
The Integration of Gene Therapy for Rare Disease Article July 30, 2022
Medical Informatics and Rare Disease: a bridge between two worlds Article July 30, 2022
Millions of people have rare diseases, including my son. Here’s how Congress can help Article July 30, 2022
Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years Article July 24, 2022
Study highlights the multibillion dollar burden of rare disease Article July 24, 2022
CONSTANT AGONY Page 3 Girl Michelle Marsh reveals desperate battle to save her daughter’s life from rare disease Article July 24, 2022
There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be Article July 24, 2022
People with Rare Diseases Need Better Healthcare Article July 24, 2022
Parents are becoming drug developers to find a cure for their children’s rare diseases Article July 24, 2022
Rare Diseases in India: ‘Orphan’ No More? Article July 16, 2022
Brad Pitt believes he has rare 'face blindness' disorder—what is it? Article July 16, 2022
Lab-grown 'mini-kidneys' unlock secrets of a rare disease Article July 9, 2022
Rare diseases: "Recognition of a disability is still complicated in Europe." Article July 9, 2022
Drilling for rare disease therapeutics Article July 9, 2022
Finding Comfort in the Rare Disease Community Article July 9, 2022
What's Up Doc? Rare skin disorder causes blistering, calls for genetic counseling Article July 1, 2022
Report finds thousands of uncounted rare diseases in challenge to current estimates Article June 19, 2022
What is Cushing's disease? Experts warn of 'rare but serious' condition Article June 11, 2022
People with rare diseases have poor health-related quality of life, study finds Article June 11, 2022
What Is Ramsay Hunt Syndrome? What to Know About Justin Bieber's Rare Disease Article June 11, 2022
The Rare Disease Report Podcast: Myelodysplastic Syndromes & Acute Myeloid Leukemia Article June 4, 2022
Is your child at higher risk of rare disease? Why more couples are going for genetic tests Article June 4, 2022
Francis Collins Urges Gene Therapy Community to Scale Efforts to Tackle Rare Diseases Article May 22, 2022
Musician with rare disease overcomes health scare Article May 22, 2022
Castleman Disease: Types, Symptoms, and Treatment Article May 22, 2022
Takeda scraps dream of getting rare disease drug to market in latest setback to Wave 1 pipeline Article May 15, 2022
Lawmakers discuss push for rare disease prevention, support Article May 15, 2022
CDER Launches New Accelerating Rare disease Cures (ARC) Program Article May 15, 2022
Cilia-free stem cells offer new path to study rare diseases Article May 8, 2022
Why Pompe Disease Can Be Mistaken for Other Genetic Diseases Article May 8, 2022
Cautions issued over prenatal genetic tests in IVF and for rare diseases Article May 1, 2022
Organizations Rally to Help Ukrainian Rare Disease Patients Article May 1, 2022
Editas Medicine Gets FDA Rare Pediatric Disease Designation for EDIT-301 Article May 1, 2022
Undiagnosed Patient Organizations Begin Leveraging RARE-X Article May 1, 2022
Drug development for ultra-rare diseases: What happens when N=1? Article April 23, 2022
A dangerous delay: Why it can take years to diagnose life-threatening rare diseases Article April 23, 2022
When it comes to the rarest of diseases, the diagnosis isn't the answer – it's just the starting point Article April 23, 2022
Myotonic Dystrophy Is a Rare, Genetic Disease—And Its Link to Ventricular Tachycardia Is Even Rarer Article April 16, 2022
American Kidney Fund Applauds Introduction of New Era for Preventing End-Stage Kidney Disease Act of 2022 Article April 16, 2022
AAAS Webinar-- Reimagining rare disease detection: Who needs to be at the table? Video April 16, 2022
People with alopecia share their stories: ‘Hair does not define any of us’ Video April 14, 2022
My baby was born with a rare congenital disease, and I didn't realize until he was 2 months old. Googling his symptoms helped me advocate for him. Article April 10, 2022
Gene-therapy gel shows promise for blistering skin disease in clinical trial Article April 7, 2022
Woman with rare illness that's seen her dislocate 'hundreds' of bones can't afford to stay at university Article April 2, 2022
Increased Education Crucial to Improving Rare Disease Care, Survey Finds Article March 27, 2022
Doctor Who Devised a Treatment for Her Rare, Painful Disease Started Clinic to Help Others Article March 27, 2022
Ascentage Pharma's MDM2-p53 Inhibitor Alrizomadlin (APG-115) Granted Rare Pediatric Disease Designation By The US FDA For The Treatment Of Neuroblastoma Article March 27, 2022
Kids with rare autoimmune disease show these symptoms before blood clots Article March 27, 2022
I've had a blood transfusion every 3 weeks since I was 2 months old due to a rare disease that can kill people in their 30s Article March 20, 2022
For people living with rare diseases, waiting on the STAT Act can be frustrating Article March 20, 2022
Science Brings Shortcut to Spotting 50 Rare Genetic Diseases Article March 13, 2022
Science Brings Shortcut to Spotting 50 Rare Genetic Diseases Article March 13, 2022
Climbing Mount Everest? A courageous dad fundraises to find cure for a muscle disease in hopes of saving son’s life. Article March 13, 2022
Report: Economic burden of rare diseases is 10 times higher than mass market diseases Article March 5, 2022
Rare Disease Symposium connects families, doctors and scientist looking for answers Article March 5, 2022
Cell research on rare disease finds new link to inflammation Article March 5, 2022
It’s a rare disease affecting 200,000 women, no cure, and no one is talking about it until now Video March 5, 2022
My Daughter's Rare Disease Was A Mystery For Years. Here's How We Finally Got A Diagnosis. Article March 5, 2022
Rare Disease Day: Parents share arduous experience, hopeful future in raising children with rare diseases Article March 5, 2022
First Gene Therapy For Tay-Sachs Disease Successfully Given To Two Children Article February 22, 2022
Early Trial Offers Hope Treating Rare 'Brittle Bone' Disease Article February 22, 2022
New FDA program could boost drug development for rare diseases Article February 22, 2022
We in the Rare Disease Community Can Learn From Each Other Article February 6, 2022
Scientists identify new features of lymphangioleiomyomatosis, a rare lung disease Article February 6, 2022
There's no cure for rare types of cystic fibrosis, but researchers are making significant advances Article February 6, 2022
Parents Lose Two Babies Weeks Apart as Child Dies of Rare Genetic Disease Article February 6, 2022
Record-breaking rapid DNA sequencing promises timely diagnosis for thousands of rare diseases Article February 6, 2022
Analysis reveals rare respiratory disease PCD is more common than previously thought Article January 29, 2022
My baby was born with disease so rare it doesn’t have a name – I’m desperate to find cure before he dies Article January 29, 2022
Parents Refuse To Take No For An Answer When Son Is Diagnosed With Incurable Disease Article January 22, 2022
Children With Rare Fatal Disease Trial World-First Treatment To Save Their Sight Article January 15, 2022
What to Know About Scleroderma, the Autoimmune Disease Bob Saget Fought for Before His Death Article January 15, 2022
Hutchinson-Gilford Progeria Syndrome: What to Know About the Rare and Fatal Genetic Disorder Article January 15, 2022
Herpes virus "likely" main cause of Multiple Sclerosis study finds Article January 15, 2022
Diagnostic odyssey: The lonely road walked by thousands of Coloradans with disorders so rare even their doctors hadn't heard of them Article January 1, 2022
The Colorado Parents Fighting the World’s Rarest Diseases Article January 1, 2022
First-ever United Nations Resolution to Increase Visibility for the 300 Million Persons Living with a Rare Disease Article December 19, 2021
Treatment in Texas: For families of kids with rare diseases, it’s a full-time job to advocate for, raise millions for research Video December 12, 2021
24-Year-Old Researches Treatment for Her Own Crippling Disease: 'I'm in a Race Against Time' Article December 5, 2021
Rare gene in Amish people helps protect their hearts Article December 5, 2021
Gene-Based Therapy for Rare Skin Disease Succeeds in Phase III Article November 30, 2021
General Hospital' star Bergen Williams dead at 62 after battling rare Wilson's disease Article November 20, 2021
New treatment for a rare genetic disease to be made available on NHS Article November 20, 2021
FDA Approves Treatment for Rare Blood Disease Article November 14, 2021
Rare Genetic Mutation in Utah Family Traced Across Continents And Over Centuries Article November 14, 2021
Hundreds of patients in gene study given rare disease diagnosis Article November 14, 2021
Whole genome sequencing improves diagnosis of rare diseases and shortens diagnostic journeys for patients Article November 14, 2021
https://www.oudaily.com/news/national-organization-for-rare-disorders-recognizes-ou-health-as-rare-disease-center-of-excellence/article_f7e54766-3db2-11ec-a63d-3f53633a7cf1.html Article November 6, 2021
National Organization for Rare Disorders recognizes OU Health as Rare Disease Center of Excellence Article November 6, 2021
CHOC and UCI Health Join New NORD Rare Disease Centers of Excellence Network, Committed to Improving Access and Care for Rare Disease Patients Article November 6, 2021
Australia's first snapshot of families at risk of blindness from rare genetic eye disease Article November 6, 2021
FDA, NIH Announce Bespoke Gene Therapy Consortium to Focus on Rare Diseases Article October 30, 2021
NIH study suggests people with rare diseases face significantly higher health care costs Article October 24, 2021
AI Rapidly Diagnoses Rare Disorders in Critically Ill Children Article October 16, 2021
Is technology the key to accelerating rare disease clinical trials? Article October 16, 2021
Rare Disease Diversity Coalition Awards $600K to Combat Disparities Article October 16, 2021
What to know about paroxysmal nocturnal hemoglobinuria (PNH) Article October 16, 2021
New stem cell source offers hope to patients with rare liver disease Article October 16, 2021
Krystal Biotech and GeneDx Announce Collaboration to Provide No-charge Genetic Testing for Patients with Suspected Dystrophic Epidermolysis Bullosa (DEB) Article October 16, 2021
FDA awards 11 clinical trial grants for rare diseases, many in children Article October 16, 2021
ChemoCentryx's drug gets U.S. FDA nod for treating rare autoimmune disease Article October 10, 2021
Actors Who Have Rare Diseases And Medical Conditions Article October 3, 2021
New Data-sharing Program Aims to Speed Innovation in Rare Diseases Article October 3, 2021
Yeast Models Provide New Insights into Neurodegenerative Diseases Article October 3, 2021
Mirum Snags First Drug Approval for Rare, Genetic Liver Disorder Article October 3, 2021
Moderna to develop mRNA therapeutic for ultra-rare disease Article September 25, 2021
Rare Disease Spotlight: Sarcoidosis Article September 25, 2021
Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness Article September 25, 2021
Rare Diseases 2021: running rare disease trials post-Covid Article September 25, 2021
MMS support program to help ultra-rare disease research Article September 19, 2021
California legislature poised to establish Rare Disease Advisory Council Article September 19, 2021
The Champion for Rare Disease Cures Article September 19, 2021
8-Year-Old Girl Is Left Paralyzed After COVID Likely Triggered Rare Disease Article September 19, 2021
New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many Article September 11, 2021
Moderna donates ultra-rare disease therapy to nonprofit founded by late Takeda R&D chief Article September 11, 2021
‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases Article August 29, 2021
BioMarin stands tall with first approved drug for rare disease that causes dwarfism Article August 29, 2021
AstraZeneca drug for rare disease shows promise in flushing copper build-up Article August 29, 2021
A father on the legacy of his son's ultra-rare disease Podcast August 29, 2021
British father begins 1,200- mile barefoot walk in Maine to raise awareness for daughter's rare disorder Video August 29, 2021
Woman hits the road to support Rare Disease Cause Video August 22, 2021
8-year-old Camryn Kellam Beating Back Blood Disease, Rare Brain Syndrome Video August 22, 2021
Parents create nonprofit to find cure for toddler's rare disease Video August 22, 2021
This Gainsville mom wants to raise awareness of rare disease with upcoming walk at Wilshire Trail Article August 17, 2021
With a nudge from AI, ketamine emerges as a potential rare disease treatment Article August 8, 2021
For two UB scientists, love means studying daughter’s rare disease Article August 8, 2021
Cecilia woman testifies to Congress about rare disease Article July 31, 2021
‘I Just Want My Kid To Have A Good Life’: Colorado Boy 1 In 40 Worldwide Diagnosed With Rare Disease Video July 31, 2021
Rare disorder offers roadmap for understanding roots of inflammatory disease Article July 31, 2021
Healx: AI-driven drug repurposing for rare disease Article July 24, 2021
NIH-funded study finds gene therapy may restore missing enzyme in rare disease Article July 18, 2021
Early Study Shows Promise of CRISPR Injection to Treat Rare Disease Article July 18, 2021
Is Artificial Intelligence The Best Solution For Diagnosing Rare Diseases? Article July 18, 2021
Community rallies around 6-year-old with rare disease. His family needs your help. Article July 11, 2021
Dying patients with rare diseases struggle to get experimental therapies Video July 11, 2021
Photographs reveal people behind rare illnesses Article July 11, 2021
After Timesia Hart Was Diagnosed With a Rare Disease, She Started a Bike Race to Inspire Others Article July 7, 2021
Legislation to Help Diagnose Children with Rare Diseases Introduced by Senators Collins, Kelly, and Menendez Article June 27, 2021
The Loneliness I Feel as the Parent of a Child With a Rare Disease Article June 20, 2021
Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines Article June 20, 2021
KC family worries cost could keep drug from helping son with rare disorder Video June 12, 2021
Columbus mom lost one daughter to a rare disease. A new drug is helping another Video June 12, 2021
Newer methods may boost gene therapy's use for more diseases Article June 5, 2021
The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price Article June 5, 2021
FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder Article June 5, 2021
Unprecedented data sharing driving new rare disease diagnoses in Europe Article June 5, 2021
Raleigh biopharma company hopes to have a new treatment for celiac disease Article June 5, 2021
Las Vegas woman writes children's book to help people with rare disease Video May 21, 2021
‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access Article May 21, 2021
Child fights for her life by taking the 'most expensive drug in the world' Video May 21, 2021
Cystic Fibrosis: One Patient’s Story, Current Treatments and Exciting New Therapies on the Horizon Article May 16, 2021
Rare Disease Companies Unite to Advocate for Life-Changing Therapies Article May 16, 2021
Moms search for lifesaving bone marrow donors for daughters with rare diseases Article May 16, 2021
Why I Hate My Kid's Birthday Article May 16, 2021
Rare diseases the next target for mRNA therapies Article May 16, 2021
Treating Polycythemia Vera Article May 16, 2021
CENTOGENE Discovers Six New Rare Diseases by Leveraging the Strength of Its Bio/Databank Article May 9, 2021
Gap in care, research and funding for rare blood disorder disproportionately affecting Black Americans Video May 9, 2021
Woman with rare muscular disease sings to exercise her lungs. Her songs inspire others to chase their dreams Video May 2, 2021
Chiesi Global Rare Diseases Announces FDA Approval of FERRIPROX® (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease Article May 2, 2021
Virtual gala to raise funds, awareness for rare neurodevelopmental disorder Article May 2, 2021
CDISC and NORD Partner to Develop Data Standards for Rare Diseases Article April 24, 2021
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US Article April 17, 2021
Detecting rare disease: Revealing the methods, motivations, and implications Article April 17, 2021
A new treatment for rare muscular disease Article April 17, 2021