Sarepta Therapeutics announced positive clinical data showing its experimental gene therapy increased expression of a missing gene in patients with an ultra-rare limb-girdle muscular dystrophy subtype (LGMD 2E) and said it plans to file for regulatory approval. The company presented the data publicly and framed the program as a potential first approved treatment for a subset of LGMD. The lead sentence: Sarepta will pursue approval after trial data showed target-gene level restoration in affected patients, a milestone in a disease area with no approved disease-modifying therapies. The company disclosed biomarker and functional readouts that underpin its filing strategy. Regulatory reviewers will evaluate efficacy signals, safety, and manufacturing consistency; payers and clinicians will monitor durability and patient selection criteria. The filing would mark a marquee approval attempt in the rare-disease gene-therapy space and could set precedents for evidence expectations in ultra-rare neuromuscular indications.