The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Europe's medicines regulator did not recommend the approval of Sarepta Therapeutics' gene therapy Elevidys for a type of rare ...

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

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

Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...

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

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...