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

Most Americans encounter the Federal Trade Commission only if they’ve been scammed: It handles identity theft, fraud, and ...

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

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