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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

Status: Recruiting
Start Date: October 2021
Intervention Type: Drug, Biological, Other
Phase: Phase 1/Phase 2
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Summary: 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)

Status: Recruiting
Start Date: November 23, 2020
Intervention Type: Other
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Summary: The primary objective of this study is to evaluate potential biomarkers of GSD III.
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