Sarepta presented data showing its experimental gene therapy increased expression of the missing gene in patients with limb girdle muscular dystrophy (LGMD) 2E and said it plans to file for regulatory approval. The dataset indicates biochemical target engagement in an ultra-rare form of muscular dystrophy where no approved therapies exist. Sarepta characterized the finding as the first step toward an approval pathway that could deliver the first treatment option for a subset of LGMD patients. The company acknowledged regulatory and clinical hurdles ahead, including the rarity of the condition and the need for robust durability and safety data. Investors and clinicians will watch for the timing and scope of Sarepta’s submission and whether regulators require additional efficacy endpoints or confirmatory trials before granting approval.