Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

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

US regulators said they are investigating the death of an 8-year-old boy who received a gene therapy from Sarepta ...

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 ...

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 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 ...

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

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 ...

Sarepta officials said the temporary halt in shipments was done to maintain a productive working relationship with regulators ...