https://www.wallstreet-online.de/nachricht/20690496-ultragenyx-announces-u-s-fda-acceptance-of-bla-resubmission-for-ux111-aav-gene-therapy-to-treat-sanfilippo-syndrome-type-a-mps-iiia
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat...
02.04.2026 - If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children...