STAT | Reporting from the frontiers of health and medicine Follow STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease The FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s. https://www.statnews.com/2026/09/17/sanfilippo-syndrome-treatment-ultragenyx-approval/ statnews.com
