Gene Therapy Death Raises Concerns for Rare Muscle Disorder Treatment

Sarepta Therapeutics Inc. reported its second patient death due to acute liver failure after receiving its gene therapy Elevidys for Duchenne muscular dystrophy. The death occurred three months after a teenage boy’s fatal reaction to the treatment, also targeting patients with this rare muscle disorder who are unable to walk.

Source: https://www.bloomberg.com/news/articles/2025-06-15/sarepta-reports-second-death-of-patient-using-its-gene-therapy