Wilson's disease

Lancet. 2007 Feb 3;369(9559):397-408. doi: 10.1016/S0140-6736(07)60196-2.

Abstract

Progressive hepatolenticular degeneration, or Wilson's disease, is a genetic disorder of copper metabolism. Knowledge of the clinical presentations and treatment of the disease are important both to the generalist and to specialists in gastroenterology and hepatology, neurology, psychiatry, and paediatrics. Wilson's disease invariably results in severe disability and death if untreated. The diagnosis is easily overlooked but if discovered early, effective treatments are available that will prevent or reverse many manifestations of this disorder. Studies have identified the role of copper in disease pathogenesis and clinical, biochemical, and genetic markers that can be useful in diagnosis. There are several chelating agents and zinc salts for medical therapy. Liver transplantation corrects the underlying pathophysiology and can be lifesaving. The discovery of the Wilson's disease gene has opened up a new molecular diagnostic approach, and could form the basis of future gene therapy.

Publication types

  • Review

MeSH terms

  • Chelating Agents / adverse effects
  • Chelating Agents / therapeutic use*
  • Copper / metabolism*
  • Copper / urine
  • Hepatolenticular Degeneration* / drug therapy
  • Hepatolenticular Degeneration* / genetics
  • Hepatolenticular Degeneration* / physiopathology
  • Humans
  • Liver Transplantation
  • Penicillamine / adverse effects
  • Penicillamine / therapeutic use*
  • Trientine / therapeutic use*

Substances

  • Chelating Agents
  • Copper
  • Penicillamine
  • Trientine