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.

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

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

Sarepta said it accepts the CHMP decision on Elevidys, while partner Roche said it will continue working with the EMA.