Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start

OBJECTIVE: To examine respiratory muscle function among late-onset Pompe disease (LOPD) patients in the Pompe Registry (NCT00231400/Sanofi Genzyme) during enzyme replacement therapy (ERT) with alglucosidase alfa by assessing the longitudinal course of forced vital capacity (FVC), prognostic factors...

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Detaylı Bibliyografya
Yayımlandı:J Neurol
Asıl Yazarlar: Stockton, David W., Kishnani, Priya, van der Ploeg, Ans, Llerena, Juan, Boentert, Matthias, Roberts, Mark, Byrne, Barry J., Araujo, Roberto, Maruti, Sonia S., Thibault, Nathan, Verhulst, Karien, Berger, Kenneth I.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Springer Berlin Heidelberg 2020
Konular:
Original Communication
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7501128/
https://ncbi.nlm.nih.gov/pubmed/32524257
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-020-09936-8
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