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...

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Detalhes bibliográficos
Publicado no:J Inherit Metab Dis
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Netherlands 2015
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Original Article
Acesso em linha: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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