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Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease
N W Barton1, R O Brady, J M Dambrosia
1Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892.
The Journal of Pediatrics
|February 1, 1992
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.