Patients with Duchenne muscular dystrophy (DMD) who received delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) showed long-term disease stabilization or slowed disease progression at 3 and 5 years of follow-up in data presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas.1,2
Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy approved for the treatment of ambulatory patients 4 years of age or older with a confirmed mutation in the DMD gene and for patients who are non-ambulatory and have a confirmed mutation in the DMD gene under accelerated approval.3
Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy approved for the treatment of DMD. Image credit: natali_mis – stock.adobe.com
Pooled…