US EPA

4835825 

Journal Article 

Review 

Clinical considerations for an effective medical therapy in Wilson's disease 

Weiss, KH; Stremmel, W 

2014 

Yes 

Annals of the New York Academy of Sciences
ISSN: 0077-8923
EISSN: 1749-6632 

1315 

81-85 

English 

24754532 

10.1111/nyas.12437 

Wilson's disease is an autosomal recessively inherited copper overload disorder that leads to hepatic and/or neurologic symptoms. More than a century after the first description of Wilson's disease, the available medical treatment options have not been standardized. The efficacy of the commonly used drugs is satisfactory for hepatic disease, but disappointing in the neurologic patients, including the risk of neurologic deterioration after the initiation of chelation therapy. An approach to overcome this problem is the careful and systematic assessment of biochemical response patterns and the quantitative monitoring of symptoms using validated rating scales. Standardized dosage strategies that address changes in copper pools might improve adherence and reduce side effects. Such an approach may reduce long-term morbidity. In this paper, we discuss considerations for an effective medical treatment and requirements for future studies in Wilson's disease.