The Food and Drug Administration approved an updated safety warning for a gene therapy treatment that restricts its use following reports of two deaths among pediatric users, the agency said in a statement on Nov. 14.
The approval was granted for Elevidys, a one-time gene therapy treatment for Duchenne muscular dystrophy (DMD) by pharmaceutical company Sarepta Therapeutics Inc. DMD renders muscle cells vulnerable to injury, leading to progressive muscle degeneration.
