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

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'SRPT brutal week continued Friday after European officials rejected the company's controversial gene therapy, Elevidys.The rejection, which was delivered to European partner ...

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