title: Imiglucerase and its use for the treatment of Gaucher's disease.
abstract: BACKGROUND
  Gaucher's disease is caused by deficient lysosomal glucocerebrosidase activity. Intravenous enzyme replacement therapy with imiglucerase (Cerezyme, Genzyme Corporation, Cambridge, MA), a recombinant human glucocerebrosidase, ameliorates systemic manifestations such as hepatosplenomegaly, anemia, thrombocytopenia and skeletal abnormalities in patients with type 1 (non-neuronopathic) and type 3 (chronic neuronopathic) Gaucher's disease.
  
  
  OBJECTIVE
  The aim of this study was to identify and comment on the current issues related to imiglucerase for Gaucher's disease based on a review of published English language literature and personal clinical experience.
  
  
  RESULTS
  The following topics were covered with respect to imiglucerase: development, pharmacokinetics, preparation and administration, efficacy, pediatrics, pregnancy, type 3 Gaucher's disease, dosing, treatment interruptions, safety and alternative pharmacological therapies.
  
  
  CONCLUSIONS
  Imiglucerase is safe and well tolerated. In addition, it corrects the hepatic, splenic, hematologic and bone abnormalities observed with types 1 and 3 Gaucher's disease effectively and enhances health-related quality of life.
authors.affiliation: University Research Foundation for Lysosomal Storage Diseases, Inc., Northwest Oncology Hematology Associates PA, 8170 Royal Palm Boulevard, Coral Springs, FL 33065, USA. boneal@winning.com
authors.forename: Neal J
authors.initials: NJ
authors.lastname: Weinreb
mesh_terms: D000328:Adult; D000818:Animals; D002648:Child; D002986:Clinical Trials as Topic; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000503:physiopathology; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000009:adverse effects / Q000378:metabolism / Q000493:pharmacokinetics; D006801:Humans; D007275:Injections, Intravenous; D011247:Pregnancy; D011788:Quality of Life
chemical_list: D005962:Glucosylceramidase; C090568:imiglucerase