First gene therapy tested in children with rare muscle disease

This early-stage study is testing the safety of a single-dose gene therapy called ATA-200 in children with a rare genetic muscle disorder called limb girdle muscular dystrophy type 2C. The therapy aims to deliver a working copy of a faulty gene directly into the body via an intravenous infusion. Researchers will carefully monitor four young patients for side effects over several years to determine if the treatment is safe enough for future studies.