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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Bibliografiska uppgifter
I publikationen:J Inherit Metab Dis
Huvudupphovsmän: 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.
Materialtyp: Artigo
Språk:Inglês
Publicerad: Springer Netherlands 2015
Ämnen:
Original Article
Länkar: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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