WILSON DISEASE
Clinical trials for WILSON DISEASE explained in plain language.
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Gene therapy could free wilson disease patients from daily pills
Disease control OngoingThis study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. Ab…
Matched conditions: WILSON DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: WILSON DISEASE
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 02:00 UTC
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Wilson disease patients sought for survey to shape gene editing research
Knowledge-focused OngoingThis study surveys 30 adults with Wilson disease to learn about their daily management, treatment adherence, and interest in gene editing clinical trials. By gathering this information, researchers hope to design better future studies for potential gene therapies targeting specif…
Matched conditions: WILSON DISEASE
Sponsor: Prime Medicine, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:31 UTC
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Tiny trial uses PET scans to peek at gene therapy for wilson disease
Knowledge-focused ENROLLING_BY_INVITATIONThis study will use special PET scans to see how gene therapy (UX704) changes copper distribution in the bodies of 5 people with Wilson disease. The goal is to understand if the therapy helps the liver handle copper better. It is an early, observational imaging study, not a test …
Matched conditions: WILSON DISEASE
Sponsor: Thomas Damgaard Sandahl • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:22 UTC