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Ultragenyx GSD1A Community Update Fall 2025
Ultragenyx GSD1A Community Update Fall 2025 DTX401 program updates and global community engagement highlights. View Community Update [...]
Fall 2025 Newsletter
Fall 2025 Newsletter AGSD Annual Conference 2025 – A Resounding Success! This past June, the AGSD community gathered in [...]
Beam 301 -STRIVING FOR A NEW POTENTIAL THERAPY FOR GSD1a
THE BEAM-301 CLINICAL STUDY A phase 1/2, multicenter, open-label, single-ascending dose study to evaluate the safety and efficacy profiles of [...]
We Need Your Voice!
EL-PFDD interactive virtual meeting on GSD1a. Living with Glycogen Storage Disease Type 1a October 9, 2025 More Information [...]
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage [...]
Introduction to Beam’s Clinical Development Program for GSD1a
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