Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease.
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.
Medical subject headings
- Gaucher Disease
- Glucosylceramidase