The natural course of non–classic Pompe’s disease; a review of 225 published cases

@article{Winkel2005TheNC,
  title={The natural course of non–classic Pompe’s disease; a review of 225 published cases},
  author={L{\'e}on P. F. Winkel and Marloes Louise Catharina Hagemans and Pieter Antoon van Doorn and M. Christa B. Loonen and Wim C. J. Hop and Arnold J. J. Reuser and PhD A. T. Van der Ploeg},
  journal={Journal of Neurology},
  year={2005},
  volume={252},
  pages={875-884}
}
Pompe’s disease is a neuromuscular disorder caused by deficiency of lysosomal acid α–glucosidase. Recombinant human α– glucosidase is under evaluation as therapeutic drug. In light of this development we studied the natural course of cases not fitting the definition of classic infantile Pompe’s disease. Our review of 109 reports including 225 cases shows a… CONTINUE READING