Sarepta Therapeutics reported data showing its experimental gene therapy increased expression of the missing gene in limb‑girdle muscular dystrophy type 2E (LGMD 2E) and said it plans to file for regulatory approval. If approved, Sarepta’s candidate would be the first therapy indicated specifically for LGMD, a collection of ultra‑rare neuromuscular diseases that cause progressive muscle weakness. The company presented biomarker and expression data to support a regulatory submission; details and broader efficacy endpoints remain limited publicly. For readers: LGMD covers genetically distinct subtypes; LGMD 2E stems from deficiency in a particular structural muscle protein and has no approved disease‑modifying therapies today.