New gene therapy hopes to stop deadly infant muscle disease

This study tests a one-time gene therapy called GB221 in infants with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The therapy delivers a working SMN1 gene to nerve cells to help them survive and function. The trial includes babies aged 2 weeks to under 12 months who have symptoms, and presymptomatic babies up to 5 months old. Researchers will monitor safety and whether the treatment improves motor milestones and reduces the need for permanent breathing support.