New Guidelines Issued for Gene Therapy Treatment in Duchenne Muscular Dystrophy

The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) have jointly published consensus guidelines on the safe and equitable administration of gene therapy for Duchenne muscular dystrophy (DMD) in Neuromuscular Disorders. The recommendations provide neurologists with a standardized framework for patient selection, institutional readiness, and monitoring practices to improve safety and consistency across treatment centers.

The guidelines were developed in collaboration with cardiologists from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION Network) and experts from 15 leading MDA and PPMD Clinical Care Centers. Drawing on clinical experience and multidisciplinary input, the statement emphasizes evidence- and practice-informed recommendations in the absence of long-term outcome data.

Key recommendations include:

• Cross-center collaboration and the delivery of multidisciplinary care
• Protocols for early identification and management of adverse events, particularly in the first 3 months post-infusion.
• Cross-center reporting mechanisms to capture and share safety data.
• Guidance on accessibility, financial support, and referral pathways to minimize disparities in treatment availability.

In a statement issued by the MDA, the organization cites the Food and Drug Administration (FDA) approval of Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA) in 2023—the first gene therapy for people with DMD—and other agents in the developmental and regulatory pipeline, along with uncertainties about long-term durability, timing of dosing, and response variability due to genetic background, as highlighting the need for these guidelines in clinical practice.

“Gene therapy offers hope for altering the course of Duchenne muscular dystrophy, but the safe and effective delivery requires specialized expertise and vigilant follow-up,” said Barry Byrne, MD, PhD, Director of the Powell Gene Therapy Center at the University of Florida, Member of the MDA Board of Directors, and Leader of the guideline development team. “These consensus guidelines are an important step toward unifying care practices, closing knowledge gaps, and supporting research that will refine these guidelines as our understanding evolves.”

Source: Wolff JM, Capocci N, Atas E, et al. Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy. Neuromuscular Disorders. 2025;0(0):106208. doi:10.1016/j.nmd.2025.106208

Muscular Dystrophy Association. Muscular Dystrophy Association and Parent Project Muscular Dystrophy announce joint consensus guidelines for safe and equitable delivery of gene therapy in Duchenne muscular dystrophy. August 23, 2025. Accessed August 23, 2025. https://www.mda.org/press-releases/mda-and-ppmd-announce-joint-consensus-guidelines.