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Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up

BACKGROUND: Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy (ERT) has been available since 2006. Effects of ERT have been shown on distance walked, pulmonary function and survival. We investigated whether it also improves quality of life and participation in d...

詳細記述

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書誌詳細
出版年:J Inherit Metab Dis
主要な著者: Güngör, Deniz, Kruijshaar, Michelle E., Plug, Iris, Rizopoulos, Dimitris, Kanters, Tim A., Wens, Stephan C. A., Reuser, Arnold J. J., van Doorn, Pieter A., van der Ploeg, Ans T.
フォーマット: Artigo
言語:Inglês
出版事項: Springer Netherlands 2015
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC4754323/
https://ncbi.nlm.nih.gov/pubmed/26531313
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-015-9889-6
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