Health
FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A
On September 17, 2026, the U.S. Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf) as the first gene therapy for pediatric patients with Sanfilippo syndrome type A. The one-time intravenous treatment uses an AAV9 vector to deliver a functional copy of the SGSH gene to cells.