Aravindhan Veerapandiyan, MD, Pediatric Neuromuscular Neurologist at Arkansas Children’s Hospital, discusses new data from the EMBARK part 2 study of Elevidys (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy (DMD).
DMD is a rare genetic, neuromuscular disease that leads to progressive muscle wasting. DMD is an x-linked disorder and primarily impacts males. The symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscles. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the DMD gene.
Delandistrogene moxeparvovec is a single-dose, adeno-associated virus (AAV) gene therapy administered via intravenous infusion. It is designed to address the disease-causing gene mutations in the DMD gene that result in a lack of dystrophin protein by targeting production of micro-dystrophin in the skeletal muscle.
New data from the EMBARK trial was presented at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. Patients included in this part 2 study had received placebo in part 1 and were aged 8 to 9 years at crossover. At one year post treatment with delandistrogene moxeparvovec, statistically significant improvements were observed including 4.75 points on North Star Ambulatory Assessment, a 6.87 second time-to-rise from floor, and a 4.76 second 10-meter walk/run compared to a control cohort. Additionally, no new safety signals were observed and micro-dystrophin expression and sarcolemmal localization were sustained from week 12 to week 64.
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To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/