Glycogen Storage Disease Type Ia
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Ultragenyx is conducting a disease monitoring program for Glycogen Storage Disease Type Ia, and the study is currently recruiting participan…
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) [Phase 3]
Phase 3 study of AAV8-mediated glucose-6-phosphatase gene transfer for Glycogen Storage Disease Type Ia has completed.
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Accuracy of Lactate Meter in GSDIa
A recruiting clinical trial evaluating the accuracy of a lactate meter in patients with Glycogen storage disease type Ia.
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CLINICAL_TRIALS research · 2025-12-15Clinical Trial Update: A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a) [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating mRNA-3745 for Glycogen storage disease type I (Ia) is ongoing, with the study conducted by Moderna and…
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CLINICAL_TRIALS other · 2025-12-15Clinical Trial Update: A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 dose-exploration study to evaluate the safety and efficacy of BEAM-301 in patients with Gly…
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CLINICAL_TRIALS trial_result · 2024-05-29Clinical Trial Update: Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
A retrospective study evaluating glucose monitoring for glycemic control in patients with Glycogen Storage Disease Type Ia has completed. Sp…
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CLINICAL_TRIALS trial_result · 2022-11-18Clinical Trial Update: Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) [Phase 1, Phase 2]
Phase 1/2 safety and dose-finding clinical trial of DTX401 (AAV8G6PC) in adults with Glycogen Storage Disease Type Ia has completed with res…
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CLINICAL_TRIALS trial_result · 2022-02-11Clinical Trial Update: Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia [Phase 1, Phase 2]
Phase 1/2 trial investigating endogenous glucose production in patients with Glycogen Storage Disease Type Ia has completed.
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CLINICAL_TRIALS research · 2014-12-18Clinical Trial Update: Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
A completed clinical trial exploring the relationship between Glycogen Storage Disease Type Ia and inflammatory bowel disease.
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FDA approval ·FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia
FDA approves the first therapy (Genglycos) for patients aged 8 years and older with glycogen storage disease type Ia (GSDIa) under accelerat…