Glycogen storage disease I
MONDO:0002413Glycogenosis due to glucose-6-phosphatase (G6P) deficiency or glycogen storage disease, (GSD), type 1, is a group of inherited metabolic diseases, including types a and b, and characterized by poor tolerance to fasting, growth retardation and hepatomegaly resulting from accumulation of glycogen and fat in the liver.
Also known as: G6P deficiency, GSD due to G6P deficiency, GSD type 1, GSD type I, GSD1, Glycogen Storage Disease Type I, glycogen storage disease I, glycogen storage disease due to G6P deficiency
13 clinical trials for this condition and its sub-types.
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Gene therapy could free GSD ia patients from constant cornstarch
Disease control CompletedThis Phase 3 trial tested a gene therapy called DTX401 in 49 people with glycogen storage disease type Ia (GSD Ia). The goal was to see if a single infusion could reduce or eliminate the need for frequent cornstarch doses to keep blood sugar stable. Participants were randomly ass…
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check
Disease control CompletedThis study checked the long-term safety of a one-time gene therapy (DTX401) in 12 adults with glycogen storage disease type Ia, a rare condition that causes dangerously low blood sugar. Researchers monitored side effects and how well the therapy helped control blood sugar levels …
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC