Publication

Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.

Journal Paper/Review - Aug 28, 2024

van Kooten Harmke A, Horton Mike C, Wenninger Stephan, Babačić Haris, Schoser Benedikt, Lefeuvre Claire, Taouagh Najib, Laforet Pascal, Segovia Sonia, Diaz-Manera Jordi, Claeys Kristl G, Mongini Tiziana, Musumeci Olimpia, Toscano Antonio, Hundsberger Thomas, Brusse Esther, van Doorn Pieter A, van der Ploeg Ans T, van der Beek Nadine A M E, European Pompe Consortium study group on outcome measures