Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A Ultragenyx’s UltraCare® program will support access, and commercial product is expected