Gene Therapy for Muscular Dystrophy Faced New Scrutiny After Second Death
A second patient death has been reported in connection with Sarepta Therapeutics’ gene therapy Elevidys, which treats muscular dystrophy. The therapy, approved in 2023 for children with a specific genetic variant of Duchenne’s muscular dystrophy, is believed to cause liver injuries in some patients. Sarepta announced it would pause shipments of the therapy for older … Read more