Sarepta Therapeutics presented data showing its experimental gene therapy increased expression of the missing gene in limb girdle muscular dystrophy 2E (LGMD2E) and said it plans to file for regulatory approval. The company aims to deliver the first approved therapy for this ultra‑rare LGMD subtype, which can cause progressive muscle weakness and loss of ambulation. Sarepta’s disclosure signals a high‑stakes regulatory push for a disease area with no approved systemic therapies. The filing will put the therapy before regulators who will weigh surrogate biomarker changes, functional endpoints, and safety signals from the clinical program. Analysts expect regulatory scrutiny given small patient populations and the heterogeneity across LGMD subtypes.