title: Deferiprone for the treatment of Friedreich's ataxia.
abstract: Friedreich's ataxia (FRDA) is a neurological disease related to a deficiency of the protein frataxin involved in iron-sulfur (Fe-S) cluster biogenesis. This leads to an increased cellular iron uptake accumulating in mitochondria, and a subsequently disturbed iron homeostasis. The detailed mechanism of iron regulation of frataxin expression is yet unknown. Deferiprone, an iron chelator that may cross the blood-brain barrier, was shown to shuttle iron between subcellular compartments. It could also transfer iron from iron-overloaded cells to extracellular apotransferrin and pre-erythroid cells for heme synthesis. Here, clinical studies on Deferiprone are reviewed in the context of alternative agents such as desferoxamine, with specific regard to its mechanistic and clinical implications.
authors.affiliation: Department of Neurology, Université Libre de Bruxelles, Hôpital Erasme, Brussels, Belgium. massimo.pandolfo@ulb.ac.be
authors.forename: Massimo
authors.initials: M
authors.lastname: Pandolfo
authors.forename: Laura
authors.initials: L
authors.lastname: Hausmann
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D000077543:Deferiprone; D004353:Drug Evaluation, Preclinical; D005621:Friedreich Ataxia / Q000188:drug therapy*; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D033862:Iron-Binding Proteins / Q000378:metabolism; D011728:Pyridones / Q000627:therapeutic use*; D013455:Sulfur / Q000378:metabolism; D000096923:Frataxin
chemical_list: D000077543:Deferiprone; D007501:Iron; D007502:Iron Chelating Agents; D033862:Iron-Binding Proteins; D011728:Pyridones; D013455:Sulfur; D000096923:Frataxin
keywords: Deferiprone; Friedreich's ataxia (FRDA); clinical study; frataxin