Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.

ABSTRACT: Delandistrogene moxeparvovec is currently the only commercially approved gene therapy for Duchenne muscular dystrophy (DMD). Herein, we report real-world data of 11 ambulatory patients with DMD, ages 4-6, treated with commercial delandistrogene moxeparvovec. Patients were prospectively and uniformly monitored for 1 year post-gene transfer for safety and motor outcomes. Nine patients experienced 15 treatment-related toxicities, and 4 required escalation of corticosteroids. Side effects included gastrointestinal symptoms (n = 7), liver enzyme abnormalities (n = 4), acute liver injury (n = 2), and troponin I elevations (n = 3). In both patients with acute liver injury, troponin I elevations occurred in close temporal association beginning 8-9 weeks post-gene transfer and were responsive to co

SUBMITTER: Keselman DJ 

PROVIDER: S-EPMC12925777 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature