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, 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 ...

Criticism of Prasad's tenure intensified around the agency's handling of a gene therapy for Duchenne muscular dystrophy from Sarepta Therapeutics. The FDA-approved therapy played a role in the death ...

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

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.

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 ...