MDA 2025: Advancing Care in DMD and SMA
Ryan Haumschild, PharmD, MS, MBA, CPEL; and Perry B. Shieh, MD, PhD, discuss how gene therapies like delandistrogene moxeparvovec for Duchenne muscular dystrophy and onasemnogene abeparvovec for spinal muscular atrophy are showing promising long-term efficacy in stabilizing motor function and potentially transforming treatment outcomes, while addressing ongoing challenges including ensuring equitable access, managing high costs, and the need for sustained durability data.
Episodes
June 16th 2025EP. 1: Advancements & Challenges in Muscular Dystrophy Treatment
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer technologies, while highlighting the ongoing challenges of identifying at-risk populations and collecting comprehensive safety and efficacy data.
June 16th 2025EP. 2: Data on Duchenne Muscular Dystrophy Treatment From MDA 2025
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North Star Ambulatory Assessment, time to rise from floor, and 10-m walk/run compared with external control groups over 2 years of treatment.
