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11769 results for "rare disease advocacy"
A mother whose daughter was diagnosed with a rare genetic disorder created an AI platform that saves families 53 hours a week and accelerates treatment research by up to 50%. Citizen Health now helps over 8,000 patients across 350 rare diseases navigate care and connect with others facing the same challenges.
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.
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.
A hospital in Indonesia built a successful rare disease registry in just two years, offering hope for similar efforts in Malaysia. The achievement could help 45 million people across Southeast Asia living with rare conditions get better care. #
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.
Patient-led groups fighting rare diseases are getting a massive boost. The Chan Zuckerberg Initiative just announced $150 million in funding to help families turn hope into real treatments.
Scientists just achieved a major breakthrough in treating two rare muscle diseases that currently have no cure. Early trials show a new delivery method gets medicine directly into muscle cells without serious side effects.
After nearly dying five times from a mystery illness, Dr. David Fajgenbaum turned to artificial intelligence to save his own life—and now he's finding treatments for thousands of other forgotten patients. His AI system has already identified potential cures for over 60 rare diseases that drug companies won't touch.
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.
After a decade of stagnation where only 5% of rare diseases had treatments, Europe is overhauling outdated regulations to bring hope to 30 million patients. New pathways are finally treating ultra-rare conditions differently than common illnesses.
A paralyzed doctor who couldn't breathe on his own made a miraculous recovery from an autoimmune disease so rare he was only the 17th documented case worldwide. Now he's raising awareness and funds for the aqua therapy that saved him.
A new AI algorithm called EvORanker is ending the decade-long "diagnostic odyssey" for families with rare diseases, identifying the correct disease-causing gene in 70% of test cases. The tool looks across evolution to spot hidden genetic patterns that traditional medicine misses.
After years without hope, Susannah Rosen received a breakthrough treatment for a rare neurodegenerative disorder that was slowly killing her brain cells. A nonprofit foundation now provides the custom-made medicine free for life to patients with ultra-rare diseases.
Scientists in Austin are developing a one-of-one therapy for baby Everett Blomstrom, who has an ultra-rare genetic disease affecting fewer than 50 people worldwide. The experimental treatment could be ready in two years and represents a breakthrough in personalized medicine for rare conditions.
Scientists are using a safer twist on gene editing to treat diseases by flipping genetic switches instead of cutting DNA. The first trial results for a rare muscular disorder just arrived, and they're opening doors for conditions from high cholesterol to inherited diseases.
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.
A new FDA pathway could cut the time to get personalized CRISPR treatments from four years to just three months, making life-saving gene editing accessible to thousands of children born with rare genetic diseases. Baby KJ Muldoon became the first person to receive this revolutionary treatment in February 2025.
A Nashville family transformed their annual Kentucky Derby party into a powerful fundraiser after their daughter was diagnosed with an incurable nerve disease. Last year alone, they raised $75,000 for research into Charcot-Marie-Tooth disease.
A pharmaceutical company has brought new hope to families affected by a rare disease by resurrecting a gene therapy program that was once thought lost. The treatment offers a second chance for patients in a community that had faced devastating setbacks.
Two AI-powered companies just received FDA approval to tackle thousands of rare diseases that have gone untreated for decades due to scientist shortages. Their breakthrough platforms can automate gene editing and drug discovery, potentially bringing personalized medicine to millions within 10 years.
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