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 ...

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 ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

Dyne Therapeutics' DYNE-251 receives FDA breakthrough status for Duchenne muscular dystrophy treatment. Read more here.

The event, held from 11 a.m. to 4 p.m. at 332 North High Street, will benefit Dayton and Blake Biesecker of Biglerville, twin brothers who were diagnosed with Duchenne muscular dystrophy at age 6 ...