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

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.

Despite regulatory setbacks, the petitioners believe that survival and other data they've collected themselves are worth ...