FDA Lifts Hold on DMD Gene Therapy for Ambulatory Patients | JQJO

The FDA recommends lifting its voluntary hold on Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD), for ambulatory patients. An investigation concluded that a previous patient death was unrelated to the treatment. The hold remains for non-ambulatory patients following two deaths in that group. The FDA emphasizes continued engagement with the DMD community.