What is Fayuvi, the first gene therapy for Sanfilippo syndrome Type A?

Fayuvi is described in the approval announcement as the first gene therapy for pediatric patients with Sanfilippo syndrome Type A, a rare inherited disorder that prevents the body from properly breaking down certain complex sugars. Gene therapy aims to deliver a working genetic instruction rather than repeatedly treating one symptom, but “first” does not mean simple or risk-free. Eligibility, timing, monitoring, and the complete FDA label must be reviewed with a specialist experienced in the disease.