- recruiting
Natural History of Wilson Disease
This study aims to better understand Wilson Disease, a rare genetic condition where too much copper builds up in the body. Researchers want to find the best ways to diagnose it and check if treatments are working. By collecting information from patients, they hope to improve future care.
United States · Germany · United Kingdom - active not recruitingPHASE1, PHASE2
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
This study is testing a new gene therapy called UX701 for adults with Wilson disease. It aims to see if UX701 is safe, works well to help control copper levels, and to find the best dose. It will compare UX701 to usual treatments.
United States · Canada · Portugal