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

Publikation

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

Wissenschaftlicher Artikel/Review - 14.06.2024

Schoser Benedikt, van der Beek Nadine A M E, Broomfield Alexander, Brusse Esther, Diaz-Manera Jordi, Hahn Andreas, Hundsberger Thomas, Kornblum Cornelia, Kruijshaar Michelle E, Laforet Pascal, Mengel Eugen, Mongini Tiziana, Orlikowski David, Parenti Giancarlo, Pijnappel W W M Pim, Roberts Mark, Scherer Thomas, Toscano Antonio, Vissing John, O'Sullivan Joe M, van Doorn Pieter A, Wenninger Stephan, van der Ploeg Ans T

Zitation

Schoser B, van der Beek N, Broomfield A, Brusse E, Diaz-Manera J, Hahn A, Hundsberger T, Kornblum C, Kruijshaar M, Laforet P, Mengel E, Mongini T, Orlikowski D, Parenti G, Pijnappel W, Roberts M, Scherer T, Toscano A, Vissing J, O'Sullivan J, van Doorn P, Wenninger S, van der Ploeg A. Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024. Eur J Neurol 2024:e16383.

Art

Wissenschaftlicher Artikel/Review (Englisch)

Zeitschrift

Eur J Neurol 2024

Veröffentlichungsdatum

14.06.2024

eISSN (Online)

1468-1331

Seiten

e16383

Kurzbeschreibung/Zielsetzung

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 recommendations for starting, switching and stopping ERT.