Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

M. Ripolone, R. Violano, D. Ronchi, S. Mondello, A. Nascimbeni, I. Colombo, G. Fagiolari, A. Bordoni, F. Fortunato, V. Lucchini, S. Simona, M. Filosto, O. Musumeci, P. Tonin, T. Mongini, S. Previtali, L. Morandi, C. Angelini, M. Mora, M. Sandri, M. Sciacco, A. Toscano, G. P. Comi, M. Moggio
  • Neuropathology and Applied Neurobiology, July 2017, Wiley
  • DOI: 10.1111/nan.12414

study of biopsies in late onset pompe cases

What is it about?

Compare before and after ERT

Why is it important?

Shows increase in GAA


Prof Corrado Angelini
IRCCS S.Camillo Hospital Foundation

Further studies on improvement

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The following have contributed to this page: Prof Corrado Angelini