Paul Melmeyer, MPP, on the Importance of Gene Therapy for MDA’s Upcoming 2025 Conference

“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.”

Each year, the Muscular Dystrophy Association (MDA), an organization dedicated to serving the community of patients with muscular dystrophies and other neuromuscular disorders, holds the MDA Clinical & Scientific Conference. The event brings together practicing clinicians, medical researchers, and members of patient advocacy groups under one roof to discuss and share ongoing research in the field. Notably, this year, the event will be held on March 16-19, 2025, in Dallas, Texas.

In early anticipation of the upcoming conference, CGTLive® has gotten in touch with Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, to learn more about the growing importance of the annual meeting to the field of gene therapy and the significance of the conference for the aforementioned interested groups. Melmeyer emphasized the importance of the conference for advocacy groups, around 40 to 50 of which are likely have a presence at the conference. Furthermore, he noted that members of these advocacy groups would convene for a meeting referred to as the Neuromuscular Advocacy Collaborative focused on public policymaking goals intended to serve their patient communities.

Melmeyer also spoke about the growing role of gene therapy in the field of neuromuscular disease, noting that investigational gene therapy candidates, Sarepta Therapeutics’ FDA approved gene therapydelandistrogene moxeparvovec-rokl (marketed as Elevidys) for Duchenne muscular dystrophy, and Novartis’ FDA approved gene therapy onasemnogene abeparvovec-xioi (marketed as Zolgensma) for spinal muscular atrophy will be major focuses at the 2025 MDA conference. He concluded by discussing what he personally is looking forward to at the upcoming conference.

Click here to register for the upcoming 2025 MDA Conference.