title: Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease.
abstract: Long-term studies of a child with Gaucher disease indicated that the response to treatment with macrophage-targeted glucocerebrosidase (glucosylceramidase) is dose dependent, and that the hematologic response precedes the skeletal response.
authors.affiliation: Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892.
authors.forename: N W
authors.initials: NW
authors.lastname: Barton
authors.forename: R O
authors.initials: RO
authors.lastname: Brady
authors.forename: J M
authors.initials: JM
authors.lastname: Dambrosia
authors.forename: S H
authors.initials: SH
authors.lastname: Doppelt
authors.forename: S C
authors.initials: SC
authors.lastname: Hill
authors.forename: C A
authors.initials: CA
authors.lastname: Holder
authors.forename: H J
authors.initials: HJ
authors.lastname: Mankin
authors.forename: G J
authors.initials: GJ
authors.lastname: Murray
authors.forename: G C
authors.initials: GC
authors.lastname: Zirzow
authors.forename: R I
authors.initials: RI
authors.lastname: Parker
mesh_terms: D000005:Abdomen / Q000473:pathology; D001842:Bone and Bones / Q000473:pathology; D002648:Child; D004305:Dose-Response Relationship, Drug; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000175:diagnosis / Q000188:drug therapy* / Q000201:enzymology; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000097:blood; D006801:Humans; D008264:Macrophages / Q000187:drug effects; D008279:Magnetic Resonance Imaging; D008297:Male
chemical_list: D005962:Glucosylceramidase