The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

Sarepta Therapeutics’ stock was soaring Tuesday after the FDA recommended lifting the pause on the company’s Duchenne ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

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

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...