Ultragenyx posts results of Phase 3 DTX401 trial in Glycogen Storage Disease Type Ia
The study enrolled 52 participants and completed primary data collection on February 20, 2024. It evaluated DTX401's ability to reduce or eliminate dependence on exogenous glucose replacement therapy and to improve glucose control quality.
Primary source
ClinicalTrials.gov results record, October 14, 2026
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Read the source: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) clinicaltrials.gov
Machine-summarized from the source above; not independently reported. How we use automation