Business Wire·Sep 25, 2026, 8:30 AM ET

Sarepta Therapeutics Announces Presentations at 2026 World Muscle Society Annual Congress

SRPT

Sarepta Therapeutics will present new data on its Duchenne muscular dystrophy portfolio at the 31st World Muscle Society Congress in Hiroshima, Japan, from Sept. 29 to Oct. 3.
Six poster presentations regarding delandistrogene moxeparvovec (ELEVIDYS) efficacy, safety, & micro-dystrophin expression will occur on Sept. 30, including studies on older ambulatory patients & early intervention.
Specific sessions cover functional outcomes in patients aged 8-12 years, 3-year post-infusion data from the EMBARK study, & pooled safety analyses from Phase 1 to Phase 3 trials.
Additional presentations include efficacy data from DMD MDX mouse models & Phase 3 results comparing golodirsen & casimersen with placebo in the ESSENCE study.
ELEVIDYS is indicated for ambulatory patients aged 4 years & older with confirmed DMD gene mutations, but is contraindicated for those with deletions in exon 8 &/or exon 9.
The therapy carries a Boxed Warning for acute serious liver injury & acute liver failure, requiring systemic corticosteroids before & after infusion, plus weekly liver monitoring for the first 3 months.
Other critical warnings include risks of myocarditis, infusion-related reactions, immune-mediated myositis, & serious infections due to immunosuppression.
Preexisting immunity to AAVrh74 is a key consideration, with ELEVIDYS not recommended for patients with anti-AAVrh74 antibody titers ≥1:400.
Most common adverse reactions reported in clinical studies include vomiting, nausea, liver injury, pyrexia, thrombocytopenia, & increased troponin-I.