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Glycogen Storage Disease Type 3 Clinical Trials
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Found 2 clinical trials
A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients With GSD III
Drug, Biological, Other
Phase 1/Phase 2
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).
Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
November 23, 2020
The primary objective of this study is to evaluate potential biomarkers of GSD III.
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