Wilson Disease
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Real World Evidence Study in Subjects With Wilson's Disease
This clinical trial update reports the completion of a real-world evidence study in subjects with Wilson's disease, sponsored by Orphalan.
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride [Phase 2]
A Phase 2 patient-preference study comparing standard of care versus once-daily trientine tetrahydrochloride for Wilson's disease has comple…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial
Prime Medicine is running a prescreening study to identify Wilson disease patients for an upcoming gene-editing clinical trial. The study is…
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease [Phase 1, Phase 2]
A Phase 1/2/3 trial of UX701 gene therapy for Wilson disease is currently active but not recruiting.
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients. [Phase 3]
A Phase 3 trial assessing once-daily trientine tetrahydrochloride as first-line treatment for Wilson's disease is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease Monitoring
A completed clinical trial evaluates exchangeable and relative exchangeable copper as alternatives to 24-hour urinary copper for monitoring …
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CLINICAL_TRIALS research · 2026-03-05Clinical Trial Update: Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study
A Yale University pilot study is examining urinary copper excretion in Wilson disease patients off treatment and is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-01-28Clinical Trial Update: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease [Phase 1, Phase 2]
A Phase I/II trial update for VTX-801 in adults with Wilson's disease, with results posted January 28, 2026.
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease [EARLY_Phase 1]
An early-phase 1 clinical trial evaluating the safety and potential efficacy of LY-M003 injections in Wilson disease is currently recruiting…
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity [NA]
This clinical trial investigates skin elasticity in Wilson disease patients treated with D-penicillamine to characterize treatment-related s…
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101) [EARLY_Phase 1]
EARLY Phase 1 clinical trial update for DSL101, an ATP7B mRNA/LNP therapy for Wilson disease, currently recruiting.
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients
A clinical trial update from Hospices Civils de Lyon investigating circadian variation of urinary copper excretion in Wilson disease patient…
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: Oral Health and Wilson's Disease: SOMAWI
This clinical trial update indicates that the SOMAWI study on oral health in Wilson disease is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: Cardiac Involvement in Wilson's Disease [NA]
A clinical trial investigating cardiac involvement in Wilson disease is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease [NA]
A clinical trial update indicates recruitment for a study measuring copper concentration in breast milk of women treated for Wilson's Diseas…
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CLINICAL_TRIALS research · 2025-12-10Clinical Trial Update: Description of Renal Involvement in Wilson's Disease
A clinical trial update focusing on renal involvement in Wilson disease, with the study not yet recruiting.
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CLINICAL_TRIALS trial_result · 2025-12-05Clinical Trial Update: Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease [NA]
A clinical trial evaluating the MEX-CD1 low-volume continuous venovenous haemodialysis device for copper extraction in Wilson disease has be…
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CLINICAL_TRIALS research · 2025-11-21Clinical Trial Update: Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map
An upcoming protocol at Assiut University in Upper Egypt is evaluating multifaceted assessments for Wilson's disease, including service inte…
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CLINICAL_TRIALS research · 2025-10-06Clinical Trial Update: Endocrine Dysfunction in Pediatric Wilson's Disease
A clinical trial sponsored by Assiut University investigating endocrine dysfunction in pediatric Wilson's disease, currently not yet recruit…
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD) [Phase 1, Phase 2]
A Phase I/II clinical trial is planned to evaluate GC310 Injection in Wilson disease. Recruitment has not yet begun.
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
An ongoing clinical trial is evaluating a gene therapy for Wilson disease, using 64Cu PET/CT imaging to assess copper metabolism.
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CLINICAL_TRIALS trial_result · 2025-08-22Clinical Trial Update: WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation) [NA]
A completed study evaluating Wilson STIMulation Deep Brain Stimulation (WILSTIM DBS) for severe dystonia in Wilson's disease, sponsored by H…
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CLINICAL_TRIALS trial_result · 2025-08-15Clinical Trial Update: Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily). [Phase 1]
A Phase 1 trial comparing a once-daily 900 mg dose of TETA 4HCl to Cuprior (trientine) in Wilson's disease has completed and posted results.
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CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: International Wilson's Disease Patient Registry (iWilson Registry)
Ongoing recruitment for the International Wilson disease Patient Registry (iWilson Registry) sponsored by Orphalan.
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EMA approval · 2025-07-02EMA Authorised: Cufence (trientine dihydrochloride) — Univar Solutions BV
The EMA authorised Cufence (trientine dihydrochloride) from Univar Solutions BV for Wilson disease (hepatolenticular degeneration). The deci…