Current Research Studies
A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-finding, and Phase 3 Clinical Study of UX701 AAV-mediated Gene Transfer for the Treatment of Wilson Disease
UX701-CL301
What is the goal of the study?
This study investigates the safety and efficacy of a gene therapy (UX701) for Wilson Disease
Who can participate in the study?
Please contact the study team listed below to learn more.