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Clinical Trial Search block Test

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

Phase: I/II
Clinicaltrials.gov ID: NCT04884815

UX701-CL301

What is the goal of the study?

This study investigates the safety and efficacy of a gene therapy (UX701) for Wilson Disease

Everyone