Zolgensma: A Revolutionary Gene Therapy for Spinal Muscular Atrophy
Zolgensma: A Revolutionary Gene Therapy for Spinal Muscular Atrophy Introduction Zolgensma (onasemnogene abeparvovec) is a groundbreaking gene therapy designed to treat spinal muscular atrophy (SMA), a severe neuromuscular disorder caused by mutations in the SMN1 gene. Developed by Novartis Gene Therapies, Zolgensma is currently the most expensive drug in the Leggi tutto