Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

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

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

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

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

The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an ...

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

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

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

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

Sarepta continues to work with regulators to complete the safety label update for Elevidys, and they are discussing an approach for risk mitigation for non-ambulatory patients.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report second quarter 2025 financial results after the Nasdaq Global Market closes on ...