Sarepta Therapeutics presented data showing its experimental gene therapy raised expression of the missing gene in limb‑girdle muscular dystrophy type 2E (LGMD2E) and announced plans to file for regulatory approval. If cleared, the therapy would be the first approved treatment for an LGMD subtype and could set a regulatory precedent for ultrarare muscular dystrophies. Company data indicate biochemical correction in treated patients; Sarepta acknowledged regulatory challenges and expected an uphill review process given small patient numbers and complex endpoints common to rare‑disease gene therapies. The filing will focus attention on trial design, durability and manufacturing scale for personalized systemic gene therapies.