Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Glycogen storage disease type 3

Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III) Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies