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

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...

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 ...

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.

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne muscular dystrophy. U.S. distribution has been paused amid safety concerns.

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 and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

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