Michael Kelly, PhD, on Continuing Progress in Muscular Dystrophy in 2024


The chief scientific officer of CureDuchenne discussed research he is looking forward to seeing in 2024.

“I think it's the best of both worlds right now. There are companies that are clinically active right now examining new ways to enhance exon skipping by targeting muscle, so you can actually improve the efficiency of exon skipping.This is an exciting time. The data that we're going to see for the remainder of this year and the first part of the next year is going to be incredibly informative for what we know about these approaches, trying to get approved therapies to patients, and how to make decisions about which ones to use.”

Although the FDA approved the first gene therapy in the muscular dystrophy field, delandistrogene moxeparvovec (Elevidys) for the treatment of Duchenne muscular dystrophy (DMD), in 2023, the field is anticipating more data on Elevidys as well as other programs in development. Recently, Sarepta Therapeutics announced data from the global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) that showed that the study had failed its primary end point.

The EMBARK study failed to reach statistical significance in its key primary end point of change in North Star Ambulatory Assessment (NSAA) scores at week 52 compared to placebo. Key secondary end points of change in time to rise (TTR) and 10-meter walk test (10MWT) compared to placebo were statistically significant across all age groups.

CGTLive spoke with Michael Kelly, PhD, chief scientific officer, CureDuchenne, to learn more about the current treatment landscape for DMD and other muscular dystrophies. He shared his anticipation to see more data coming in the next year on novel therapies for DMD.

Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0
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