The Significance of Gene Therapy in Neuromuscular Medicine at the 2025 MDA Conference: Paul Melmeyer, MPP
Noah Stansfield
Prior to the 2025 MDA Conference, the vice president of Public Policy & Advocacy at the Muscular Dystrophy Association talked about the increasing interest in gene therapy in the field of neuromuscular diseases.
“The field is learning at lightspeed pace about gene therapies... It's really quite an amazing time to be a clinician in neuromuscular diseases, learning more about how to administer gene therapies and how these gene therapies that are approved by the FDA actually work for those with neuromuscular conditions.”
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Melmeyer also addressed the expanding role of gene therapy in neuromuscular disease, pointing to investigational gene therapy candidates and FDA-approved treatments like delandistrogene moxeparvovec-rokl (Elevidys; Sarepta) for Duchenne muscular dystrophy and onasemnogene abeparvovec-xioi (Zolgensma; Novartis) for spinal muscular atrophy as key topics at the 2025 conference. He concluded by sharing his personal excitement for the upcoming event.
Registration for the 2025 MDA Conference is now open! The meeting is set to be held at the Hilton Anatole, Dallas, Texas, from March 16 to 19, 2025. To register and for more information, head to
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