Enzyme replacement therapy and fatigue in adults with Pompe disease

Deniz Güngör,Juna de Vries,Esther Brusse,Michelle Kruijshaar,Linda van den Berg,Wim Hop,Arnold Reuser,Pieter van Doorn,Marloes Hagemans,Iris Plug,Ans van der Ploeg

Molecular Genetics and Metabolism（2013）

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摘要

•We studied an international cohort of 163 adult Pompe patients on enzyme therapy.•Levels of fatigue decreased significantly during treatment.•Fatigue decreased in women, older patients and those with shorter disease duration.•The mechanisms underlying this effect require further investigation.

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关键词

ERT,FSS,MRC,FVC,HADS

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