Prasad and FDA Commissioner Marty Makary had asked Sarepta to cease shipping its treatment for Duchenne muscular dystrophy. At the FDA, Prasad will resume his role as head of CBER, but it’s unclear ...

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 ...

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

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary ...

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

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

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

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.

Deklan Locke, whose nickname is Flash, has a rare illness, Duchenne muscular dystrophy, which causes progressive muscle ...