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138 results for "rare disease"
A mother is running the London Marathon to fund research for gene therapies that could save her daughter from kidney failure. Her eight-year-old was diagnosed with a rare, incurable form of nephrotic syndrome that currently has no treatment options.
Children with rare genetic growth disorders grew twice as fast after starting a groundbreaking treatment, offering hope where few options existed before. The therapy worked especially well for kids whose condition directly matched the drug's target.
OpenAI just released GPT-Rosalind, an AI designed to help scientists discover life-saving drugs faster. With 300 million people worldwide waiting for treatments for rare diseases, this tool could cut years off the typical 10-15 year drug development timeline.
Pharmaceutical company Roche announced a new clinical trial that could bring a life-changing gene therapy for Duchenne muscular dystrophy to young patients across Europe. The move comes after extensive feedback from families and regulators who want to see this treatment option become available.
Doctors are finally cracking the code on rare T-cell lymphomas that have resisted treatment for decades. A team of researchers across eight major U.S. medical centers is mapping personalized treatments that could transform survival rates.
After 20 years without new treatments, the FDA approved Avlayah, the first therapy designed to treat both physical and neurological symptoms of Hunter syndrome. The breakthrough offers hope to families battling this rare disease that causes progressive organ damage and early death.
UK regulators just approved olezarsen, the first treatment in years for familial chylomicronemia syndrome, a rare genetic condition that can cause life-threatening pancreatic inflammation. Clinical trials showed patients reduced dangerous fat levels by a third while experiencing fewer emergency attacks.
People with a rare genetic disorder that causes dangerous fat buildup in their blood now have a breakthrough treatment that cuts triglyceride levels by nearly a third. The UK just approved olezarsen for adults with familial chylomicronemia syndrome, a condition that can trigger life-threatening pancreas inflammation.
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.
Researchers at the University of Michigan have discovered what causes a rare condition that makes walking so painful patients crawl instead. An FDA-approved cream has already shown promise in early trials.
When physician-scientist David Fajgenbaum was told there was nothing left to try for his rare disease, he started asking a different question. Together with social entrepreneur Kiah Williams, he's proving that turning hope into action creates real change.
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A Colorado scientist's breakthrough research is transforming treatment for children with a devastating rare disease, bringing relief after years of pain. Two patients are already showing remarkable improvement from a new therapy approach.
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.
An emergency physician discovered his son had a rare genetic condition, transforming both his approach to medicine and his understanding of empathy. Dr. Joseph D'Orazio now says raising Gabe made him a better doctor and taught him what patients truly need.
A promising therapy for fragile X syndrome, a leading genetic cause of autism, is moving to human trials after securing funding from strategic partners. The treatment could also help patients with other rare brain disorders.
When doctors couldn't help her son's rare brain disorder, Amber Freed learned genetics, coordinated researchers worldwide, and raised millions to create the first gene therapy for his condition. Maxwell, now 8, became the first person ever to receive the groundbreaking treatment.
A mother who raised $1.3 million to fund a controversial treatment for her son's fatal muscle disease just saw clinical trial results that stunned experts. The experimental drug uses a counterintuitive approach: fixing broken genes by breaking them a little more.
Children with a devastating form of epilepsy may soon have new hope thanks to promising trial results from an experimental treatment. Praxis Precision Medicines announced their drug elsunersen successfully reduced seizures in early testing for SCN2A-related epilepsy.
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 bipartisan bill would make insurance cover prescribed medical nutrition that keeps nearly half a million Americans healthy. Families currently pay thousands out of pocket for doctor-prescribed treatments insurance companies refuse to cover.
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