On Saturday 12 July, Claire Ellis, 34, a teacher from Doncaster set off from her home to cycle 100km in support of her son ...

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.

A teenager living with a severe and degenerative condition is hoping to retain his independence in the face of the cruel ...

K.S.R.A. Praneeth, an 18-year-old battling Duchenne Muscular Dystrophy, competed in the World IPCA Chess Championship in Goa, ...

Last month Claire Ellis, aged 34, a teacher from Doncaster, set off from her home to cycle 100km in support of her son Declan ...

SAN DIEGO, Aug. 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today ...

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

Vinay Prasad is returning to his role overseeing vaccine, gene therapy and blood product regulation at the U.S. Food and Drug ...

With a strong launch underway for a bladder cancer gene therapy, Ferring Pharmaceuticals is finding the kind of commercial ...

The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

Vinay Prasad, MD, MPH, is resuming leadership of the FDA center that regulates vaccines and biotech therapies, an HHS ...