title: Enzyme replacement therapy in pediatric patients with Gaucher disease: what should we use as maintenance dosage?
abstract: BACKGROUND
  No consensus exists on the minimal dose of enzyme replacement therapy (ERT) effective to maintain therapeutic goals in pediatric Gaucher patients.
  
  
  OBJECTIVE
  Evaluate the efficacy of low dosage ERT to maintain treatment goals.
  
  
  RESULTS
  Six patients had a maintenance dose of 30-35U/kg/month. All patients, with the exception of one L444P/L444P homozygote, maintained therapeutic goals.
  
  
  CONCLUSIONS
  A low maintenance dose may be adequate in most pediatric patients. L444P homozygotes may require a higher maintenance dosage.
authors.affiliation: Division of Medical Genetics, Department of Pediatrics, CHU Sainte-Justine, University of Montreal, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, Que. H3T1C5, Canada.
authors.forename: Catherine
authors.initials: C
authors.lastname: Brunel-Guitton
authors.forename: Georges-Etienne
authors.initials: GE
authors.lastname: Rivard
authors.forename: Jacques
authors.initials: J
authors.lastname: Galipeau
authors.forename: Nathalie
authors.initials: N
authors.lastname: Alos
authors.forename: Marie-Claude
authors.initials: MC
authors.lastname: Miron
authors.forename: Roxane
authors.initials: R
authors.lastname: Therrien
authors.forename: Grant
authors.initials: G
authors.lastname: Mitchell
authors.forename: Guy
authors.initials: G
authors.lastname: Lapierre
authors.forename: Marie
authors.initials: M
authors.lastname: Lambert
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000235:genetics; D005962:Glucosylceramidase / Q000235:genetics / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008297:Male
chemical_list: D005962:Glucosylceramidase