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

来源：

JOURNAL OF PEDIATRICS | 1992年 / 120卷 / 02期

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

页码：277 / 280

页数：4

共 12 条

[1] REPLACEMENT THERAPY FOR INHERITED ENZYME DEFICIENCY - MACROPHAGE-TARGETED GLUCOCEREBROSIDASE FOR GAUCHERS-DISEASE
BARTON, NW
BRADY, RO
DAMBROSIA, JM
DIBISCEGLIE, AM
DOPPELT, SH
HILL, SC
MANKIN, HJ
MURRAY, GJ
PARKER, RI
ARGOFF, CE
GREWAL, RP
YU, KT
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 1991, 324 (21) : 1464 - 1470
[2] THERAPEUTIC RESPONSE TO INTRAVENOUS INFUSIONS OF GLUCOCEREBROSIDASE IN A PATIENT WITH GAUCHER DISEASE
BARTON, NW
FURBISH, FS
MURRAY, GJ
GARFIELD, M
BRADY, RO
[J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1990, 87 (05) : 1913 - 1916
[3] BEUTLER E, 1990, BLOOD, V76, pA177
[4] REPLACEMENT THERAPY FOR INHERITED ENZYME DEFICIENCY - USE OF PURIFIED GLUCOCEREBROSIDASE IN GAUCHERS-DISEASE
BRADY, RO
PENTCHEV, PG
GAL, AE
HIBBERT, SR
DEKABAN, AS
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 1974, 291 (19) : 989 - 993
[5] SPHINGOLIPIDOSES
BRADY, RO
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 1966, 275 (06) : 312 - &
[6] KAY AC, 1991, CLIN RES, V39, pA326
[7] Ludwig J, 1979, CURRENT METHODS AUTO, P676
[8] NAIR KR, 1964, STATISTICS MATH BIOL, P119
[9] ASPECTS OF MAXIMUM LIKELIHOOD ESTIMATION OF LOGISTIC GROWTH FUNCTION
OLIVER, FR
[J]. JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION, 1966, 61 (315) : 697 - &
[10] PENTCHEV PG, 1975, J MOL MED, V1, P73

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