Sarepta Therapeutics reported data showing its experimental gene therapy raised levels of the missing protein in patients with limb‑girdle muscular dystrophy (LGMD) 2E and said it plans to seek regulatory approval. If cleared, it would be the first approved treatment for a form of LGMD. Company disclosures framed the data as biomarker evidence of target engagement rather than long‑term functional outcomes; Sarepta acknowledged an uphill regulatory path given the rarity and heterogeneity of LGMD disorders. The filing intent signals confidence in the therapy’s biological activity and a push to establish a regulatory precedent for ultra‑rare muscular dystrophies. Stakeholders will watch forthcoming submissions for clinical endpoints, comparator data, and agreed surrogate measures that regulators may accept for accelerated or conditional pathways.