Sarepta Therapeutics Unveils New DMD Research at WMS 2024

Sarepta Therapeutics Presents Latest Research on Duchenne Muscular Dystrophy at WMS 2024

Cambridge, Mass., Oct. 9, 2024 – Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, is set to present multiple studies at the World Muscle Society (WMS) 2024 conference. The presentations will cover groundbreaking research on Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), showcasing the latest advancements and clinical findings.

Key Presentations and Findings:

• 391P: A cross-sectional analysis on the agreement and accuracy of ambulatory definitions in DMD will be discussed from 8:30 to 9:30 a.m. EDT.

• 94P: The JOURNEY study, detailing the natural history of LGMDs R3-R5, will highlight baseline characteristics of the study cohort at 11:15 a.m. EDT.

• 423P: Primary results from the Phase 3 EMBARK trial will reveal the safety and efficacy of delandistrogene moxeparvovec versus placebo in DMD patients.

• 424P: Findings from the Phase 1B ENDEAVOR trial will discuss micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy in a broad DMD population.

• 425P: A five-year outcome study on delandistrogene moxeparvovec in DMD patients from a Phase 1/2a study will be presented.

• 428P: Cardiac MRI outcomes from EMBARK Part 1 will be shared, focusing on DMD patients treated with delandistrogene moxeparvovec.

For a comprehensive view of the WMS 2024 program, visit WMS 2024 Program.

About Sarepta Therapeutics

Sarepta Therapeutics is dedicated to engineering precision genetic medicine for rare diseases. With a robust pipeline of over 40 programs, Sarepta leads in the development of treatments for DMD and LGMDs through gene therapy, RNA, and gene editing. More information can be found at Sarepta Therapeutics.

Forward-Looking Statements

This release includes forward-looking statements concerning Sarepta’s research and development programs, which involve risks and uncertainties. Factors that may impact these statements include regulatory decisions, clinical trial results, and other risks detailed in Sarepta’s SEC filings. Investors should not place undue reliance on these forward-looking statements, and Sarepta does not commit to updating them, except as required by law.