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

Juna M. de Vries, Nadine Anna Maria Elisabeth van der Beek, Wim C. J. Hop, Francois P. J. Karstens, John H. Wokke, Marianne de Visser, Baziel G. M. van Engelen, Jan B. M. Kuks, Anneke J. van der Kooi, Nicolette C. Notermans, Catharina G. Faber, Jan J. G. M. Verschuuren, Michelle E. Kruijshaar, Arnold J. J. Reuser, Pieter A. van Doorn, Ans T. van der Ploeg*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Original languageEnglish
Pages (from-to)73
JournalOrphanet Journal of Rare Diseases
Volume7
DOIs
Publication statusPublished - 26 Sep 2012

Keywords

  • Pompe disease
  • Glycogen storage disease type II
  • OMIM number 232300
  • Acid alpha-glucosidase
  • Alglucosidase alfa
  • Enzyme replacement therapy
  • Lysosomal storage disorder
  • Muscle strength
  • Lung function

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