The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

The U.S. FDA is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta (SRPT) Therapeutics gene therapy for Duchenne ...

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

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 ...