Rare Disease, Pediatrics, Gene Ther

Imagine hearing the words: "Your child has a genetic disease that is likely to be fatal." For parents of infants diagnosed with Spinal Muscular Atrophy (SMA) Type 1 , this was once a devastating and hopeless reality. But today, a revolutionary treatment is rewriting that story. Its name is Zolgensma (onasemnogene abeparvovec-xioi) , and it’s not just a drug—it’s a one-time gene therapy offering a chance at a future. But what exactly is this "miracle" drug, how does it work, a