Gene therapy hope for kids with rare muscle-wasting disease

This early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a working copy of the missing gene. Participants must be able to walk and have no antibodies against the virus used to deliver the gene.