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

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

Citation

van Kooten H, Horton M, Wenninger S, Babačić H, Schoser B, Lefeuvre C, Taouagh N, Laforet P, Segovia S, Diaz-Manera J, Claeys K, Mongini T, Musumeci O, Toscano A, Hundsberger T, Brusse E, van Doorn P, van der Ploeg A, van der Beek N, European Pompe Consortium study group on outcome measures. Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale. Eur J Neurol 2024; 31:e16397.

Type

Journal Paper/Review (English)

Journal

Eur J Neurol 2024; 31

Publication Date

Aug 28, 2024

Issn Electronic

1468-1331

Pages

e16397

Brief description/objective

The Rasch-Built Pompe-Specific Activity (R-PAct) scale is a patient-reported outcome measure specifically designed to quantify the effects of Pompe disease on daily life activities, developed for use in Dutch- and English-speaking countries. This study aimed to validate the R-PAct for use in other countries.