\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the 30th<\/sup> Annual Congress of the World Muscle Society (WMS) 2025 Congress, taking place Oct. 7-11, 2025, in Vienna, Austria.<\/p>\n
\nSarepta presentations will include results from several studies in the delandistrogene moxeparvovec clinical development program, as well as a real-world evidence study of pulmonary function in advanced-stage patients with Duchenne muscular dystrophy treated with casimersen. Further, Sarepta will present results from the EMERGENE phase 3 study on the expression of SGCB and safety following bidridistrogene xeboparvovec treatment in patients with LGMD2E\/R4.<\/p>\n
\nIn addition to studies to be presented by Sarepta, multiple independent studies on delandistrogene moxeparvovec will be presented, including an abstract on the exploratory use of sirolimus prophylaxis to help mitigate the potential for acute liver injury in patients receiving delandistrogene moxeparvovec. The results presented in this abstract add to our understanding of how prophylactic use of sirolimus could potentially be part of an enhanced immunosuppressive regimen intended to reduce the risk of liver injury associated with infusion of AAV-mediated gene therapy.<\/p>\n
\n\u201cOur presentations at WMS span important milestones across our programs, including safety and micro-dystrophin expression in a larger cohort of 3- to 4-year-olds with Duchenne when treated with gene therapy, a late-breaker on considerations from an interdisciplinary expert committee in mitigating and managing acute liver injury in patients treated with delandistrogene moxeparvovec, and evidence of pulmonary stabilization in patients treated with the PMO casimersen,\u201d said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. \u201cAs leaders in genetic medicine for neuromuscular conditions, these data expand our understanding of the safety, efficacy and transformative potential of our portfolio, with the goal of ultimately improving patient care.\u201d<\/p>\n
Sarepta Podium Presentations <\/b>(*Denotes encore presentation):<\/p>\n
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