Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients

The aim of this study was to compare the long‐term outcome of classic infantile Pompe patients treated with 20 mg/kg alglucosidase alfa every other week (eow) to those treated with 40 mg/kg/week, and to study the additional effect of immunomodulation. Six patients received 20 mg/kg eow and twelve 40...

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Vydáno v:J Inherit Metab Dis
Hlavní autoři: Poelman, Esther, van den Dorpel, Jan J. A., Hoogeveen‐Westerveld, Marianne, van den Hout, Johanna M. P., van der Giessen, Lianne J., van der Beek, Nadine A. M. E., Pijnappel, W. W. M. Pim, van der Ploeg, Ans T.
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley & Sons, Inc. 2020
Témata:
Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7689828/
https://ncbi.nlm.nih.gov/pubmed/32506446
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12268
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