Start, switch and stop (triple‐S) criteria for enzyme replacement therapy of late‐onset Pompe disease: European Pompe Consortium recommendation update 2024

Schoser, Benedikt; A. M. E. van der Beek, Nadine; Broomfield, Alexander; Brusse, Esther; Díaz‐Manera, Jordi; Hahn, Andreas; Hundsberger, Thomas; Kornblum, Cornelia; E. Kruijshaar, Michelle; Laforêt, Pascal; Mengel, Eugen; Mongini, Tiziana; Orlikowski, David; Parenti, Giancarlo; Pim Pijnappel, W.W.M.; Roberts, Mark; Scherer, Thomas; Toscano, António; Vissing, John; M. P. van den Hout, Johanna; A. van Doorn, Pieter; Wenninger, Stephan; T. van der Ploeg, Ans

doi:10.1111/ene.16383

Public

Start, switch and stop (triple‐S) criteria for enzyme replacement therapy of late‐onset Pompe disease: European Pompe Consortium recommendation update 2024

Published in European Journal of Neurology • Jun 14, 2024

Authors:

Benedikt Schoser

Nadine A. M. E. van der Beek

Alexander Broomfield

Abstract

BACKGROUND AND PURPOSE: Two novel enzyme replacement therapies (ERTs), studied in phase 3 trials in late-onset Pompe patients, reached marketing authorization by the European Medicines Agency in 2022 and 2023. The European Pompe Consortium (EPOC) updates and extends the scope of the 2017 recommendat...

Subject

Enzyme replacement therapy

Medicine

Magnetic resonance imaging

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