WILSON DISEASE

This early-phase trial tests a single-dose gene therapy (LY-M003) in 18 adults and children with Wilson disease, a rare genetic condition causing copper buildup. The treatment delivers a working gene to liver cells to help remove excess copper. The main goals are checking safety …

This study looks at how Wilson's disease affects dental health. Researchers will compare tooth decay and gum health in people with two forms of Wilson's disease (liver vs. nervous system) and healthy volunteers. They will also check if copper levels in saliva can show how well tr…

This study is creating a large database to collect information about people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will track patients' health, treatments, and outcomes over time. The goal is to better understand these conditions and improv…

This study looks at whether a shorter, more convenient urine collection (8 hours) can give the same information as the standard 24-hour collection for people with Wilson disease. Wilson disease causes copper buildup in the body, and doctors monitor treatment by measuring copper i…

This study looks at how D-Penicillamine, a common treatment for Wilson's disease, affects the skin. Researchers will measure skin elasticity in 120 people—both patients on the drug and healthy volunteers—using a special mold and laser. The goal is to better understand and eventua…

This study looks at whether a urine test can be used to monitor Wilson Disease instead of a blood test. About 30 adults with stable Wilson Disease will temporarily stop their medication under close supervision. Researchers will collect daily urine and blood samples for 4 days to …