Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: Consensus recommendations

被引：77

作者：

Charrow, J

Andersson, HC

Kaplan, P

Kolodny, EH

Mistry, P

Pastores, G

Prakash-Cheng, A

Rosenbloom, BE

Scott, CR

Wappner, RS

Weinreb, NJ

机构：

[1] Childrens Mem Hosp, Div Genet, Chicago, IL 60614 USA

[2] Northwestern Univ, Feinberg Sch Med, Dept Pediat, Chicago, IL 60611 USA

[3] Tulane Univ, Sch Med, Dept Pediat, New Orleans, LA 70118 USA

[4] Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA

[5] Univ Penn, Philadelphia, PA 19104 USA

[6] NYU, Sch Med, Dept Neurol, New York, NY USA

[7] Yale Univ, Sch Med, Dept Med, New Haven, CT 06510 USA

[8] Mt Sinai Sch Med, Dept Human Genet, New York, NY USA

[9] Cedars Sinai Med Ctr, Dept Hematol & Oncol, Los Angeles, CA 90048 USA

[10] Univ Washington, Sch Med, Dept Pediat, Seattle, WA 98195 USA

[11] James Whitcomb Riley Hosp Children, Dept Pediat, Indianapolis, IN 46202 USA

[12] James Whitcomb Riley Hosp Children, Dept Med & Mol Genet, Indianapolis, IN 46202 USA

[13] Indiana Univ, Indianapolis, IN 46204 USA

[14] Univ Hosp, Dept Hematol, Tamarac, FL USA

来源：

JOURNAL OF PEDIATRICS | 2004年 / 144卷 / 01期

关键词：

D O I：

10.1016/j.jpeds.2003.10.067

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

[No abstract available]

引用

页码：112 / 120

页数：9

共 71 条

[1]

Alterini R, 1996, HAEMATOLOGICA, V81, P282

[2]

[Anonymous], 1985, PEDIATRICS, V76, P317

[3] Enzymatic and molecular diagnosis of Gaucher disease [J].

Barranger, JA ;

Rice, E ;

Sakallah, SA ;

Sansieri, C ;

Mifflin, TE ;

Cooper, DL .

CLINICS IN LABORATORY MEDICINE, 1995, 15 (04) :899-&

[4] DOSE-DEPENDENT RESPONSES TO MACROPHAGE-TARGETED GLUCOCEREBROSIDASE IN A CHILD WITH GAUCHER DISEASE [J].

BARTON, NW ;

BRADY, RO ;

DAMBROSIA, JM ;

DOPPELT, SH ;

HILL, SC ;

HOLDER, CA ;

MANKIN, HJ ;

MURRAY, GJ ;

ZIRZOW, GC ;

PARKER, RI .

JOURNAL OF PEDIATRICS, 1992, 120 (02) :277-280

[5] REPLACEMENT THERAPY FOR INHERITED ENZYME DEFICIENCY - MACROPHAGE-TARGETED GLUCOCEREBROSIDASE FOR GAUCHERS-DISEASE [J].

BARTON, NW ;

BRADY, RO ;

DAMBROSIA, JM ;

DIBISCEGLIE, AM ;

DOPPELT, SH ;

HILL, SC ;

MANKIN, HJ ;

MURRAY, GJ ;

PARKER, RI ;

ARGOFF, CE ;

GREWAL, RP ;

YU, KT .

NEW ENGLAND JOURNAL OF MEDICINE, 1991, 324 (21) :1464-1470

[6] THERAPEUTIC RESPONSE TO INTRAVENOUS INFUSIONS OF GLUCOCEREBROSIDASE IN A PATIENT WITH GAUCHER DISEASE [J].

BARTON, NW ;

FURBISH, FS ;

MURRAY, GJ ;

GARFIELD, M ;

BRADY, RO .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1990, 87 (05) :1913-1916

[7]

Belmatoug Nadia, 1995, Seminars in Hematology, V32, P33

[8]

BEUTLER E, 1990, BLOOD, V76, P646

[9]

BEUTLER E, 1971, AM J HUM GENET, V23, P62

[10]

BEUTLER E, 1994, J CLIN INVEST, V93, P1288

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