Kevin Flanigan, MD, on the Impact of Neuromuscular Disease Gene Therapy Approvals
The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.
“The real game changer in our field was the approval of a viral gene therapy for spinal muscular atrophy, which absolutely has changed the natural history of that disease. Now, in Duchenne muscular dystrophy, we have the prospect of a similar scale of advances. Although, interestingly, we have many more approaches to meaningfully work toward reexpression of the dystrophin gene. So, it's been great at this meeting to hear the latest on each of these, and to be involved in presenting some of that data as well.”
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.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
