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

Barton, N W; Brady, R O; Dambrosia, J M; Doppelt, S H; Hill, S C; Holder, C A; Mankin, H J; Murray, G J et al. · J Pediatr · 1992

case_report · Level V

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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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