Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

BACKGROUND: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscular disorder, is of promising but variable efficacy. We investigated whether it alters the course of disease, and also identified potential prognostic factors. METHODS: Patients in this open-label sing...

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Hauptverfasser: de Vries, Juna M, van der Beek, Nadine AME, Hop, Wim CJ, Karstens, Francois PJ, Wokke, John H, de Visser, Marianne, van Engelen, Baziel GM, Kuks, Jan BM, van der Kooi, Anneke J, Notermans, Nicolette C, Faber, Catharina G, Verschuuren, Jan JGM, Kruijshaar, Michelle E, Reuser, Arnold JJ, van Doorn, Pieter A, van der Ploeg, Ans T
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2012
Schlagworte:
Research
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3519647/
https://ncbi.nlm.nih.gov/pubmed/23013746
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-7-73
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