Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study

BACKGROUND: Though enzyme-replacement therapy (ERT) with alglucosidase alfa has significantly improved the prospects for patients with classic infantile Pompe disease, some 50 % of treated infants do not survive ventilator-free beyond the age of 3 years. We investigated whether higher and more frequ...

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Vydáno v:J Inherit Metab Dis
Hlavní autoři: van Gelder, C. M., Poelman, E., Plug, I., Hoogeveen-Westerveld, M., van der Beek, N. A. M. E., Reuser, A. J. J., van der Ploeg, A. T.
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Netherlands 2016
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4851694/
https://ncbi.nlm.nih.gov/pubmed/26768149
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-015-9912-y
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