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

Power soccer brought Dana Parrott freedom; now she’s on a mission to bring the sport to Connecticut. Earlier this year, ...

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

Vinay Prasad, previously critical of U.S. COVID-19 policies, has returned to his role managing vaccine and gene therapy regulation at the FDA, days after his departure. His leadership saw controversy ...

WASHINGTON — Vinay Prasad is returning to the Food and Drug Administration to resume his role overseeing vaccine, gene ...

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.

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

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

After decades of research, gene therapy has been approved for Duchenne Muscular Dystrophy, a degenerative muscle disease diagnosed each year in about 1 in 3,500 newborns, almost all boys.

Deflazacort, a newly FDA-approved drug for Duchenne muscular dystrophy, has an $89,000-a-year price tag. It has been put on hold because of an uproar over drug prices.

Duchenne Muscular Dystrophy may no longer be a death sentence. Oct. 17, 2012— -- Although he made it to 29 years old with Duchenne muscular dystrophy, Jason Williams was not expected to ...