Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease

N W Barton; R O Brady; J M Dambrosia; S H Doppelt; S C Hill; C A Holder; H J Mankin; G J Murray; G C Zirzow; R I Parker

doi:10.1016/s0022-3476(05)80444-5

Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease

J Pediatr. 1992 Feb;120(2 Pt 1):277-80. doi: 10.1016/s0022-3476(05)80444-5.

Authors

N W Barton¹, R O Brady, J M Dambrosia, S H Doppelt, S C Hill, C A Holder, H J Mankin, G J Murray, G C Zirzow, R I Parker

Affiliation

¹ Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892.

PMID: 1735829
DOI: 10.1016/s0022-3476(05)80444-5

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.

Publication types

Case Reports

MeSH terms

Abdomen / pathology
Bone and Bones / pathology
Child
Dose-Response Relationship, Drug
Follow-Up Studies
Gaucher Disease / diagnosis
Gaucher Disease / drug therapy*
Gaucher Disease / enzymology
Glucosylceramidase / administration & dosage*
Glucosylceramidase / blood
Humans
Macrophages / drug effects
Magnetic Resonance Imaging
Male

Substances

Glucosylceramidase