GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
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One-Shot gene therapy aims to free patients from constant cornstarch dependence
Disease control CompletedThis study tested a one-time gene therapy injection called DTX401 for people with Glycogen Storage Disease Type Ia (GSDIa), a rare genetic disorder. The main goal was to see if the therapy could safely reduce or eliminate the need for patients to constantly eat cornstarch to keep…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Gene therapy patients monitored for years in rare disease study
Disease control CompletedThis study followed 12 adults with a rare genetic liver disorder (GSDIa) for 4 to 6 years after they received a single dose of an experimental gene therapy. The main goal was to check for any long-term safety issues from the treatment. Researchers also monitored whether the thera…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC