WILSON DISEASE
Clinical trials for WILSON DISEASE explained in plain language.
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Gene therapy trial aims to restore copper control in wilson disease
Disease control OngoingThis study tests a new gene therapy called UX701 in 82 adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if a single dose can safely help the body regulate copper and reduce the need for daily medications. Participants will be monitore…
Matched conditions: WILSON DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 06:50 UTC
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Researchers seek wilson disease patients to shape future gene editing trials
Knowledge-focused OngoingThis study aims to better understand how people with Wilson disease manage their condition and what influences their decision to join clinical research. About 30 adults with confirmed Wilson disease will complete surveys about their symptoms, treatments, diet, and interest in a f…
Matched conditions: WILSON DISEASE
Sponsor: Prime Medicine, Inc. • Aim: Knowledge-focused
Last updated May 26, 2026 06:51 UTC
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Gene therapy for wilson disease: a closer look with PET scans
Knowledge-focused ENROLLING_BY_INVITATIONThis study uses special PET scans to see how gene therapy (UX704) changes copper levels in the liver and body of people with Wilson disease. Only 5 patients who already received the gene therapy will join. The goal is to understand if the therapy helps the liver handle copper bet…
Matched conditions: WILSON DISEASE
Sponsor: Thomas Damgaard Sandahl • Aim: Knowledge-focused
Last updated May 11, 2026 20:53 UTC