WILSON DISEASE
Clinical trials for WILSON DISEASE explained in plain language.
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One-Shot gene therapy aims to free patients from lifelong Copper-Control drugs
Disease control OngoingThis study is testing a single-dose gene therapy called UX701 for adults with Wilson Disease, a condition where the body cannot properly process copper. The main goals are to see if the treatment is safe and if it can help the body regulate copper on its own, which could reduce o…
Matched conditions: WILSON DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC
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Scientists track copper with scans after gene therapy
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to see how a new gene therapy affects copper in the body of people with Wilson disease. It uses special PET scans to track where copper goes and how it leaves the body. Only 5 adults who are already receiving the gene therapy in a separate main study can participa…
Matched conditions: WILSON DISEASE
Sponsor: Thomas Damgaard Sandahl • Aim: Knowledge-focused
Last updated Mar 10, 2026 12:53 UTC