Kevin Flanigan, MD, on the Impact of Neuromuscular Disease Gene Therapy Approvals

With the approval of Zolgensma for spinal muscular atrophy in 2019, and several gene therapies for muscular dystrophy currently in development, there has been a large amount of discussion about the potential impact of these treatments among neuromuscular disease experts.

Kevin Flanigan, MD, Director of the Center for Gene Therapy at Nationwide Children's Hospital, gave several talks focused on gene therapy in neuromuscular disease at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.

In an interview with CGTLive™’s sister publication, NeurologyLive™, Flanigan spoke about the impact of Zolgensma’s approval and the potential future impact of gene therapies that are currently in the pipeline for Duchenne muscular dystrophy (DMD). He noted that it is a very exciting time to be a neuromuscular physician, compared to just a few years ago when there were not so many prospects for meaningful, lifechanging therapies on the horizon.

Flanigan also discussed several important challenges that remain in this space. He noted that the safety and efficacy profiles of the gene therapies in development for DMD are not yet entirely clear and that it will be important to assess the risk-to-benefit ratio of each individually. Additionally, he highlighted the importance of doing earlier genetic testing to identify patients with DMD who could potentially receive gene therapies and discussed limitations of the standard genetic analysis that need to be addressed.