Emerging treatments

Trientine tetrahydrochloride

Trientine tetrahydrochloride, an alternative salt formulation to trientine hydrochloride, has been approved by the US Food and Drug Administration for the treatment of adult patients with stable Wilson's disease who are de-coppered and tolerant to penicillamine. Trientine tetrahydrochloride is not substitutable on a milligram-per-milligram basis with other trientine products. One open-label phase 3 trial of 53 adults with stable Wilson's disease concluded that trientine tetrahydrochloride was non-inferior to penicillamine for maintenance therapy.[61]

Gene therapy

Gene therapies for Wilson's disease are in phase 1/2 clinical trials.[62][63]

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