A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)
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 - UX053-CL101 A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics ...
Glycogen Storage Disease Type III (GSD III) MedDRA version: 20.1;Level: PT;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Glycogen Storage Disease Type III (GSD III) MedDRA version: 20.1;Level: PT;Classification code 10053 ...