Sarepta Therapeutics to Showcase Significant Advances in Neuromuscular Research at WMS 2025

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), renowned for its pioneering efforts in precision genetic medicine aimed at rare diseases, has announced its participation in the upcoming 30th Annual Congress of the World Muscle Society (WMS) 2025, scheduled to take place from October 7 to 11 in Vienna, Austria. The event will serve as a platform to unveil groundbreaking data from several key studies within Sarepta's neuromuscular portfolio.

A major focus of Sarepta's presentations will be the delandistrogene moxeparvovec clinical development program, particularly its safety and efficacy in treating Duchenne muscular dystrophy (DMD). The findings presented will encompass results from multiple studies, including:

Sarepta will also discuss a real-world evidence study examining pulmonary function in advanced-stage DMD patients treated with casimersen. This research aims to provide insights into the treatment's impact on respiratory health, a critical aspect of care for individuals with muscular dystrophies.

Results from the EMERGENE phase 3 study will be presented, showcasing the expression of SGCB and the safety of bidridistrogene xeboparvovec treatment in patients with limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4). This research represents a significant advancement in the understanding of gene therapy for LGMD.

In addition to its own presentations, Sarepta will feature independent studies related to delandistrogene moxeparvovec. Among these is an abstract focusing on the use of sirolimus prophylaxis to reduce the risk of liver injury associated with AAV-mediated gene therapies. This study underscores the growing body of evidence surrounding the safety profiles of gene therapies and their long-term implications.

Louise Rodino-Klapac, Ph.D., President of Research & Development and Technical Operations at Sarepta, expressed her enthusiasm about the upcoming presentations, stating, “Our 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... These data expand our understanding of the safety, efficacy and transformative potential of our portfolio, with the goal of ultimately improving patient care.”

Sarepta's contributions include both podium and poster presentations throughout the congress, with key timings as follows:

Sarepta Therapeutics' participation in the WMS 2025 congress marks a significant opportunity to share vital research findings that could reshape the landscape of treatment for neuromuscular diseases. As the company continues to lead with its innovative gene therapies, the insights generated from these studies may hold the key to enhancing patient outcomes and advancing the field of precision medicine. Investors and stakeholders are encouraged to monitor Sarepta's developments as the congress approaches, with further details available on the company's website post-event.