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NewsCLINICALTRIALS.GOVSunday, March 15, 2026 · March 15, 2026

Wilson Disease Gene Therapy Enters Clinical Trials

WHY IT MATTERS

Could replace lifelong chelation therapy. Gene therapy expanding into metabolic diseases.

First gene therapy trial for Wilson disease began enrolling, aiming to restore ATP7B protein expression.

Read the original at ClinicalTrials.gov
Gene TherapyDrug

Related conditions

Wilson disease