Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress

Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of dystrophin restoration and its impact in Duchenne CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb.

26, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 8 - 11, 2026, in Orlando, Florida. At MDA 2026, Sarepta will present new and ongoing evidence across its rare neuromuscular portfolio, including commercially available gene therapy and exon-skipping treatments in Duchenne muscular dystrophy.

Presentations include a late-breaking oral presentation on delandistrogene moxeparvovec gene therapy from the Phase 3 EMBARK study (Part 1) up to three years post-infusion compared with a matched external control. Another abstract will feature caregiver-reported impressions from the Phase 3 EMBARK study through two years of follow-up, offering complementary perspectives on treatment impact beyond clinician-reported and performance-based outcomes.

Additionally, a safety analysis of several delandistrogene moxeparvovec clinical studies with up to 7.5 years of patient follow-up will be presented; pooled data will include treatment-related adverse events that most commonly occurred within the first 60 days post-infusion. Sarepta will also present data from across its exon skipping franchise, including Phase 3 results from ESSENCE for golodirsen and casimersen.

Also, a new real-world analysis that explores survival in patients treated with exon skipping medicines will be presented, as well as interim real-world findings from the Phase 4 EVOLVE study describing long-term safety and loss of ambulation as observed in clinical practice. “At MDA we’re sharing data that reinforce dystrophin restoration as a foundational therapy and its ability to slow Duchenne disease progression over time,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta.

“We want to bring forward a data-driven view of treatment experience in clinical and real-world settings, including longer-term functional outcomes, pooled safety learnings, and caregiver-reporter perspectives that provide complementary insights into treatment effect in a subset of patients treated in EMBARK.

Our goal is to ensure clinicians and families have the information they need to make treatment decisions with confidence.” Sarepta Podium Presentation: Sarepta Poster Presentations (*Denotes encore presentation): The full MDA 2026 program is available here: https://www.mdaconference.org.

Sarepta abstracts and presentations will be available on Sarepta.com in the Events & Presentations section following the MDA embargo. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.

We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.

Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This press release contains forward-looking statements.