Recombinant human α-glucosidase from rabbit milk in Pompe patients

被引：279

作者：

Van den Hout, H

Reuser, AJJ

Vulto, AG

Loonen, MCB

Cromme-Dijkhuis, A

Van der Ploeg, AT

机构：

[1] Univ Rotterdam Hosp, Sophia Childrens Hosp, Dept Pediat, Div Neonatol, NL-3015 GJ Rotterdam, Netherlands

[2] Univ Rotterdam Hosp, Div Pediat Cardiol, Rotterdam, Netherlands

[3] Univ Rotterdam Hosp, Dept Child Neurol, Rotterdam, Netherlands

[4] Univ Rotterdam Hosp, Hosp Pharm, Rotterdam, Netherlands

[5] Erasmus Univ, Dept Clin Genet, NL-3000 DR Rotterdam, Netherlands

来源：

LANCET | 2000年 / 356卷 / 9227期

关键词：

D O I：

10.1016/S0140-6736(00)02533-2

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass Index decreased significantly. We recommend early treatment. Long-term effects are being studied.

引用

页码：397 / 398

页数：2

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