The Significance of Gene Therapy in Neuromuscular Medicine at the 2025 MDA Conference: Paul Melmeyer, MPP

“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.”

This content was originally published by our sister publication, CGTLive.

Each year, the Muscular Dystrophy Association (MDA) hosts an annual meeting dedicated to research and clinical updates for individuals with muscular dystrophies and other neuromuscular disorders. This event gathers clinicians, medical researchers, and patient advocacy groups to exchange insights and discuss ongoing initiatives in the field. In 2025, the conference will take place from March 16-19 in Dallas, Texas.

In anticipation of the event, CGTLive^® interviewed Paul Melmeyer, MPP, executive vice president of public policy and advocacy at MDA, to explore the growing significance of the conference in the realm of gene therapy and its impact on various stakeholders. In the conversation, Melmeyer highlighted the importance of the conference for advocacy groups, with around 40 to 50 expected to participate. He also noted that these groups will convene at the Neuromuscular Advocacy Collaborative to discuss public policy objectives that support their patient communities.

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 www.mdaconference.org