title: Current and future therapy in haemochromatosis and Wilson's disease.
abstract: There have been several new developments in the treatment of iron and copper overload disorders, such as haemochromatosis, thalassaemia and Wilson's disease. Clinical trials of orally administered iron chelators, both as monotherapy and in combination with deferoxamine, are in progress around the world. Several new chelators are now being introduced in clinical trials. Future therapies for iron overload may comprise of oral iron binding agents capable of preventing dietary iron absorption from the diet. The characterisation of specific iron transporters such as the divalent metallic transporter and ferroportin may hold promise for the development of 'smart' compounds capable of blocking iron transport. Several new agents are now available for the management of Wilson's disease, including trientine, zinc and tetrathiomolybdate. This review, will discuss the pathogenesis, and current and future therapies for iron and copper overload disorders.
authors.affiliation: Hepatobiliary Program, Childrens Hospital and Regional Medical Center, and University of Washington, Seattle, WA 98195, USA.
authors.forename: Karen F
authors.initials: KF
authors.lastname: Murray
authors.forename: Dickson
authors.initials: D
authors.lastname: Lam
authors.forename: Kris V
authors.initials: KV
authors.lastname: Kowdley
mesh_terms: D000284:Administration, Oral; D003300:Copper / Q000378:metabolism; D004359:Drug Therapy, Combination; D006432:Hemochromatosis / Q000188:drug therapy* / Q000235:genetics; D006527:Hepatolenticular Degeneration / Q000188:drug therapy* / Q000235:genetics; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: D007502:Iron Chelating Agents; D003300:Copper; D007501:Iron