Patient Dies After Receiving DMD Gene Therapy

March 19, 2025

Sarepta Therapeutics reported Tuesday that a patient died from acute liver failure (ALF) following treatment with delandistrogene moxeparvovec-rokl (Elevidys), an FDA-approved gene therapy for Duchenne muscular dystrophy (DMD).

While acute liver injury is a known potential side effect of AAV-mediated gene therapies and is included in the therapy’s prescribing information, Sarepta stated that ALF leading to death "represents a severity of acute liver injury not previously reported" for delandistrogene moxeparvovec, which has been used to treat more than 800 patients to date. The company also noted that the patient had a recent cytomegalovirus (CMV) infection, which the treating physician identified as a possible contributing factor.

Sarepta has notified health authorities, clinical investigators and prescribing physicians and plans to update the prescribing information to reflect this event.