Today, the Food and Drug Administration approved a new gene therapy for Spinal Muscular Atrophy. SMA Type 1 is the number one genetic killer of children 2 and under. This first-time gene therapy, developed here at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story. | Nationwide Children's Hospital

Historic Breakthrough: Gene Therapy Slows Huntington’s Disease - For the first time in history, Huntington’s disease has been successfully treated. A groundbreaking UK–US gene therapy trial has slowed the illness by 75%, targeting the toxic protein at its root. Delivered through a single brain infusion, this therapy spares neurons, preserves daily function, and offers real hope to families facing generations of suffering. Published results mark a turning point in medicine, with researchers calli