CAMBRIDGE, Mass.– Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a leader in precision genetic medicine for rare diseases, will present new data from its neuromuscular portfolio at the 30th Annual World Muscle Society (WMS) Congress, scheduled for Oct. 7–11 in Vienna, Austria.
The company’s presentations will feature results from studies in the delandistrogene moxeparvovec gene therapy program and a real-world evidence study of pulmonary function in advanced-stage Duchenne muscular dystrophy patients treated with casimersen. Sarepta will also present findings from the phase 3 EMERGENE study evaluating SGCB expression and safety outcomes following treatment with bidridistrogene xeboparvovec in patients with LGMD2E/R4.
Additional independent studies on delandistrogene moxeparvovec will be highlighted at the congress, including an abstract examining the exploratory use of sirolimus prophylaxis to mitigate the potential risk of acute liver injury associated with AAV-mediated gene therapy.
“Our presentations at WMS highlight critical progress across our programs,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations at Sarepta. “These include safety and micro-dystrophin expression data in a larger cohort of young Duchenne patients, late-breaking insights from an interdisciplinary committee on managing acute liver injury, and evidence of pulmonary stabilization with casimersen. Together, these data advance our understanding of the safety, efficacy, and transformative potential of our portfolio, with the ultimate goal of improving care for patients living with neuromuscular diseases.”
