Treatment of Wilson disease
Wilson disease is a very treatable condition. With proper therapy, disease progress can be halted and often times symptoms can be improved. Treatment is aimed at removing excess accumulated copper and preventing its reaccumulation. Treatment for Wilson disease is a lifelong process. Patients may become progressively sicker from day to day, so immediate treatment can be critical. Treatment delays may cause irreversible damage.
Chelation therapy drugs approved for treating Wilson disease include penicillamine (Cuprimine® and Depen®), trientine dihydrocholoride (Syprine®) and trientine tetrahydrochoride (Cuvrior®). These drugs act by chelation or binding of copper, causing its increased urinary excretion.
Zinc acetate is approved for treating Wilson disease under the brand names (Galzin®) in the U.S. and (Wilzin®) in Europe. Zinc acts by inducing metallothionein and blocking the absorption of copper in the intestinal tract. This action both depletes accumulated copper and prevents its reaccumulation.
Patients with severe hepatitis or liver failure may require a liver transplant. Patients being investigated or treated for Wilson disease should be cared for by specialists in Wilson disease or by specialists in consultation with their primary physicians. Stopping treatment completely will result in death, sometimes as quickly as within three months. Decreasing dosage of medications also can result in unnecessary disease progression.
Wilson disease is treatable with effective, safe medications leading to a normal life expectancy. Routine monitoring and staying on medication ae both critical to a patient’s long-term health. However, medication adherence is difficult in patients who are asymptomatic or have mild symptoms. These patients often do not see a cause and effect relationship.
Patient assistance programs are available for Wilson disease patients taking Cuvrior, Cuprimine, Syprine and Galzin. These programs provide information on getting prescriptions or assistance in covering the cost of the medication. Details can be found here: https://wilsondisease.org/living-with-wilson-disease/patient-assistance/



