title: [Pharmacological and clinical profiles of miglustat (Brazaves(®)) for the treatment of Niemann-Pick type C disease].
authors.affiliation: Teruo.Kitatani@actelion.com
authors.forename: Teruo
authors.initials: T
authors.lastname: Kitatani
authors.forename: Shuya
authors.initials: S
authors.lastname: Takahashi
authors.forename: Satoru
authors.initials: S
authors.lastname: Ikenoya
mesh_terms: D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D000818:Animals; D002118:Calcium / Q000378:metabolism; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D004791:Enzyme Inhibitors / Q000494:pharmacology* / Q000627:therapeutic use*; D005964:Glucosyltransferases / Q000037:antagonists & inhibitors; D006801:Humans; D008247:Lysosomes / Q000378:metabolism; D051379:Mice; D052556:Niemann-Pick Disease, Type C / Q000188:drug therapy* / Q000503:physiopathology
chemical_list: D004791:Enzyme Inhibitors; D017485:1-Deoxynojirimycin; C059896:miglustat; D005964:Glucosyltransferases; C023246:ceramide glucosyltransferase; D002118:Calcium