Childhood Pompe disease: clinical spectrum and genotype in 31 patients

C. I. van Capelle, J. C. van der Meijden, J. M. P. van den Hout, J. Jaeken, M. Baethmann, T. Voit, M. A. Kroos, T. G. J. Derks, M. E. Rubio-Gozalbo, M. A. Willemsen, R. H. Lachmann, E. Mengel, H. Michelakakis, J. C. de Jongste, A. J. J. Reuser, A. T. van der Ploeg*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Original languageEnglish
Article number65
JournalOrphanet Journal of Rare Diseases
Volume11
DOIs
Publication statusPublished - 18 May 2016

Keywords

  • Pompe disease
  • Childhood
  • Clinical spectrum
  • Genotype
  • Natural course

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