European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience

A. T. van der Ploeg, M. E. Kruijshaar, A. Toscano, P. Laforêt, C. Angelini, R. H. Lachmann, S. I. Pascual Pascual, M. Roberts, K. Rösler, T. Stulnig, P. A. van Doorn, P. Y. K. Van den Bergh, J. Vissing, B. Schoser
  • European Journal of Neurology, May 2017, Wiley
  • DOI: 10.1111/ene.13285

Presents an expert consensus at European level of results of ERT in late onset Pompe disease

What is it about?

Adult onset Pompe patients are a difficult group to treat a consensus was reached on criteria for ERT

Why is it important?

ERT is a rather expensive treatment and independent consensus is important

Perspectives

Prof Corrado Angelini
IRCCS S.Camillo Hospital Foundation

Further therapies can be evaluated by the consortium.

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http://dx.doi.org/10.1111/ene.13285

The following have contributed to this page: Prof Corrado Angelini