Winter 2026 Ultragenyx Update n February, Ultragenyx announced the U.S. FDA granted Priority Review for the Biologics License Application [...]

Ultragenyx Update: DTX401 BLA Accepted The U.S. FDA has granted Priority Review for the Biologics License Application (BLA) for our [...]

Ultragenyx GSD1A Community Update Fall 2025 DTX401 program updates and global community engagement highlights. View Community Update [...]

Fall 2025 Newsletter AGSD Annual Conference 2025 – A Resounding Success! This past June, the AGSD community gathered in [...]

THE BEAM-301 CLINICAL STUDY A phase 1/2, multicenter, open-label, single-ascending dose study to evaluate the safety and efficacy profiles of [...]

EL-PFDD interactive virtual meeting on GSD1a. Living with Glycogen Storage Disease Type 1a October 9, 2025 More Information [...]