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. 2006 Feb 28;66(4):581-3.
doi: 10.1212/01.wnl.0000198776.53007.2c.

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

M L C Hagemans  1 , W J C Hop, P A Van Doorn, A J J Reuser, A T Van der Ploeg
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Course of disability and respiratory function in untreated late-onset Pompe disease

M L C Hagemans et al. Neurology. .

Abstract

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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