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Thursday, September 17, 2026

Ultragenyx wins FDA full approval for gene therapy, first U.S. treatment for Sanfilippo

 

Ultragenyx wins FDA full approval for FAYUVI (UX111) gene therapy, first U.S. treatment for Sanfilippo syndrome type A

  • Ultragenyx also receives a Priority Review Voucher in connection with the FAYUVI (UX111) approval.
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