Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease.
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.
Barton
N W
NW
Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892.
Brady
R O
RO
Dambrosia
J M
JM
Doppelt
S H
SH
Hill
S C
SC
Holder
C A
CA
Mankin
H J
HJ
Murray
G J
GJ
Zirzow
G C
GC
Parker
R I
RI
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
D005962:Glucosylceramidase