DOSE-DEPENDENT RESPONSES TO MACROPHAGE-TARGETED GLUCOCEREBROSIDASE IN A CHILD WITH GAUCHER DISEASE

被引:55
作者
BARTON, NW
BRADY, RO
DAMBROSIA, JM
DOPPELT, SH
HILL, SC
HOLDER, CA
MANKIN, HJ
MURRAY, GJ
ZIRZOW, GC
PARKER, RI
机构
[1] NINCDS, BIOMETRY & FIELD STUDIES BRANCH, BETHESDA, MD 20892 USA
[2] NIH, DEPT DIAGNOST RADIOL, BETHESDA, MD 20892 USA
[3] NIH, CTR CLIN, DEPT CLIN PATHOL, BETHESDA, MD 20892 USA
[4] MASSACHUSETTS GEN HOSP, DEPT ORTHOPED SURG, BOSTON, MA 02114 USA
关键词
D O I
10.1016/S0022-3476(05)80444-5
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
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.
引用
收藏
页码:277 / 280
页数:4
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