Posted by Michael Wonder on 21 Dec 2024
Ultragenyx submits biologics license application to the US FDA for UX111 AAV gene therapy for the treatment of Sanfilippo syndrome type A
19 December 2024 - If approved, UX111 would be the first approved therapy in the US for Sanfilippo syndrome type A.
Ultragenyx today announced the submission of a biologics license application to the US FDA seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A.
