FDA Approves First-Ever Treatment for Sanfilippo Syndrome, Bringing New Hope to Children With a Fatal Rare Disease
The Food and Drug Administration has approved the first treatment for Sanfilippo syndrome type A, marking a historic breakthrough for families affected by an ultra-rare genetic disease that progressively destroys children’s neurological abilities and often leads to death in adolescence. The therapy, called Fayuvi, was developed by Ultragenyx Pharmaceutical and is intended for pediatric patients […]
