Sarepta Therapeutics reported data showing its experimental gene therapy raised levels of a missing protein in limb girdle muscular dystrophy 2E (LGMD2E) and signaled plans to file for regulatory approval — a potential first approval in the LGMD family. Sarepta presented the data as evidence of biochemical restoration in an ultra‑rare neuromuscular disorder. In parallel, Genethon reported long‑term stabilization in a small Duchenne muscular dystrophy program and intends pediatric trials and broader development. Both updates underscore continued progress and regulatory ambition in rare‑disease gene replacement programs despite small cohorts and the usual durability and safety questions that accompany systemic gene therapies.