Disease severity in children and adults with Pompe disease related to age and disease duration

被引：143

作者：

Hagemans, MLC

Winkel, LPF

Hop, WCJ

Reuser, AJJ

Van Doorn, PA

Van der Ploeg, AT

机构：

[1] Erasmus MC Sophia, Dept Pediat, Div Metab Dis & Genet, NL-3015 GJ Rotterdam, Netherlands

[2] Erasmus MC, Dept Biostat & Epidemiol, Rotterdam, Netherlands

[3] Erasmus MC, Dept Clin Genet, Rotterdam, Netherlands

[4] Erasmus MC, Dept Neurol, Rotterdam, Netherlands

来源：

NEUROLOGY | 2005年 / 64卷 / 12期

关键词：

D O I：

10.1212/01.WNL.0000165979.46537.56

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Information about 255 children and adults with Pompe disease was gathered through a questionnaire. Disease severity was associated with disease duration and not with age; an early manifestation of the disease implied earlier wheelchair or ventilator dependency. The patient group under age 15 included a subgroup with a more severe and rapid course of the disease. They require more intensive follow-up and early intervention, before irreversible damage has occurred.

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收藏

页码：2139 / 2141

页数：3

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