Sarepta Therapeutics reported data showing its experimental gene therapy increased expression of the missing gene in limb‑girdle muscular dystrophy type 2E (LGMD2E) and said it plans to file for regulatory approval. If approved, the therapy would be the first treatment for any LGMD subtype, marking a potential milestone for a family of ultra‑rare muscle disorders. Company disclosures highlighted biomarker improvements and a safety profile consistent with prior gene‑therapy programs, but executives acknowledged that regulators will require robust efficacy and durability data given the rarity and heterogeneity of LGMD presentations. Investors and clinicians flagged potential obstacles, including small trial sizes and the complexity of demonstrating clinically meaningful functional benefit in heterogeneous patient cohorts, but also noted the high unmet need in LGMD that could support expedited regulatory pathways.