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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Veröffentlicht in:J Neurol
Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Springer Berlin Heidelberg 2020
Schlagworte:
Original Communication
Online Zugang: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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