Course of disability and respiratory function in untreated late-onset Pompe disease

被引：67

作者：

Hagemans, MLC

Hop, WJC

Van Doorn, PA

Reuser, AJJ

van der Ploeg, AT

机构：

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

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

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

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

来源：

NEUROLOGY | 2006年 / 66卷 / 04期

关键词：

D O I：

10.1212/01.wnl.0000198776.53007.2c

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Fifty-two untreated patients with late-onset Pompe disease completed questionnaires about their clinical condition and level of handicap at baseline and at 1-year ( n = 41) and 2-year follow-ups ( n = 40). During this period, declines in functional activities, respiratory function, handicap, and survival were recorded on a group level. This study illustrates the progressiveness of late-onset Pompe disease and indicates the need for close clinical follow-up of both children and adults with this disorder.

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页码：581 / 583

页数：3

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