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13858 results for "rare disease advocacy"
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.
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.
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.
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.
Researchers diagnosed with rare diseases are channeling their pain into purpose, studying their own conditions to develop treatments that could save their lives and thousands of others. Their personal stakes are driving breakthroughs where traditional medicine failed.
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.
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.
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.
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.
For 40 years, treating a rare genetic lung disease meant weekly IV infusions at a clinic. Now a pill that blocks 90% of the damaging enzyme just secured a nearly half-billion dollar deal to reach American patients.
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.
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.
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