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

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

N W Barton  1 , 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
Affiliations
Case Reports

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

N W Barton et al. J Pediatr. 1992 Feb.

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.

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