GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
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Gene therapy may free GSDIa patients from constant cornstarch
Disease control CompletedThis study tested a one-time gene therapy (DTX401) in 49 people with glycogen storage disease type Ia, a rare condition that requires frequent cornstarch to prevent dangerously low blood sugar. The goal was to see if the treatment could reduce or eliminate the need for cornstarch…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 11:33 UTC
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Gene therapy for rare metabolic disease shows promise in Long-Term safety check
Disease control CompletedThis study followed 12 adults with glycogen storage disease type Ia (GSDIa) who had received a single dose of gene therapy (DTX401) in an earlier trial. The main goal was to see if the treatment was safe over the long term. Researchers also checked how well it helped control bloo…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 11:27 UTC