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17261 results for "rare disease treatment"
Children with a rare genetic liver disorder that often leads to transplant now have their first treatment to slow disease progression. After 20 years of research, Japanese doctors turned an existing drug into a life-changing option for young patients with PFIC.
Patients with a rare liver disease finally have relief from debilitating itching that robbed them of sleep and quality of life. The FDA just approved the first treatment specifically designed to ease this painful symptom that affected thousands.
After a difficult year, biotech company Sarepta Therapeutics is bouncing back with early clinical trial results showing two new muscular dystrophy treatments appear safe and effective. The promising data offers hope for patients with rare muscle-wasting diseases who desperately need new treatment options.
A California space company just partnered with a major pharmaceutical firm to manufacture impossible-to-make drugs in orbit. The collaboration could unlock breakthrough treatments for rare lung diseases by 2026.
The FDA just approved the first treatment for Menkes disease, a rare genetic disorder that has claimed countless young lives. Copper histidinate could help babies with this devastating condition finally have a fighting chance.
Scientists at the University of British Columbia have created the first topical gene therapy that corrects faulty DNA directly in human skin, offering hope for patients with painful genetic skin disorders who currently have no cure. The breakthrough treatment could eventually help millions with conditions ranging from rare inherited diseases to common eczema.
Patient groups from 10 countries just joined forces to speed up treatments for SYNGAP1, a rare genetic disorder affecting children worldwide. The new collective will coordinate research funding, clinical trials, and treatment access across continents.
Families facing Alexander disease, a rare and devastating neurological disorder, now have hope after the FDA approved the first-ever treatment for the condition. The new drug showed remarkable results in helping both children and adults walk better and maintain motor skills.
A Philadelphia nonprofit founded by a doctor who survived a rare disease just landed $76 million to help millions of patients find treatments hiding in plain sight. Every Cure's AI tool searches thousands of existing drugs to find new uses for rare diseases that lack treatments.
A doctor who survived a rare disease now uses artificial intelligence to find unexpected treatments that help patients like Kaila Mabus, who was told she might not survive. His AI system tests 75 million possible drug-disease combinations in minutes.
For two years, parents suctioned their son's airway every five minutes to keep him alive. Then an experimental treatment changed everything.
Pearl Jam's Eddie Vedder and Canadian director Matt Finlin created a documentary following the race to cure epidermolysis bullosa, a devastating skin disease affecting children worldwide. The Netflix film captures breakthrough research that could unlock treatments for thousands of rare diseases.
A Yale scientist is discovering why some patients with myasthenia gravis don't improve with standard treatments, opening doors to personalized therapies. Her groundbreaking work could help doctors match the right medicine to each patient's unique disease pattern.
A new AI-powered company is helping families with ultra-rare diseases develop personalized treatments that big pharma overlooks. Founded by a rare disease patient, Nome has already guided more than 10 genetic medicine programs from diagnosis to clinical trials.
For two years, parents suctioned their son's airway every five minutes to keep him alive. A new treatment changed everything in just weeks.
The FDA has accepted a groundbreaking treatment for Alexander disease for priority review, offering hope to families battling a rare neurological condition that has no approved therapies. If approved by September, zilganersen would become the first medicine ever available for this devastating illness.
Children and adults with a rare genetic disease that required eating every few hours can now receive a one-time gene therapy treatment. The FDA approval marks the first treatment to address the root cause of glycogen storage disease type Ia.
The Jack McGovern Coats' Foundation is launching a groundbreaking effort to create the first validated animal model for Coats' Disease, a rare condition that can cause childhood blindness. This research tool could accelerate treatment development for thousands of children worldwide.
A Colorado scientist's decade of detective work just gave hope to families battling Blau syndrome, a genetic disease affecting fewer than 1,000 Americans. Two patients are already seeing remarkable results.
A new medication just became the first therapy to directly target the damaged cells causing a serious kidney disease with no approved treatments. The breakthrough offers real hope for patients with FSGS, a rare condition that often leads to kidney failure.
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