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 ...

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

The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...

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

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

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 ...

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

Sarepta Therapeutics said the Food and Drug Administration informed the company it can lift a voluntary pause on shipments of Elevidys for ambulatory patients with Duchenne. The company said Monday ...

The company can again ship the Duchenne gene therapy for patients who can still walk, following a one-week pause triggered by ...

Sarepta Therapeutics shares are halted in extended trading Monday after the U.S. Food and Drug Administration (FDA) ...