FDA approves Fayuvi, the first treatment for Sanfilippo syndrome type A
techkahwa.net | 18 September 2026 Ultragenyx says the US Food and Drug Administration has approved Fayuvi, a one-time gene therapy for the neurologic effects of Sanfilippo syndrome type A in children. STAT reports that it is the first FDA-approved treatment for the disease, a fatal inherited condition that attacks the brain in childhood. What happened … Read more