Sarepta Therapeutics said new clinical results showed its experimental gene therapy increased expression of the missing protein in an ultra‑rare form of limb‑girdle muscular dystrophy (LGMD2E) and that the company plans to file for approval. The data, presented by Sarepta, support a first‑in‑class regulatory submission for an indication that currently lacks approved therapies. Sarepta will face regulatory scrutiny on durability, safety and manufacturing scale as it prepares its application.