Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease.
作者信息
Barton N W, Brady R O, Dambrosia J M, Doppelt S H, Hill S C, Holder C A, Mankin H J, Murray G J, Zirzow G C, Parker R I
机构信息
Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892.
出版信息
J Pediatr. 1992 Feb;120(2 Pt 1):277-80. doi: 10.1016/s0022-3476(05)80444-5.
PMID:1735829
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.
摘要
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