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H. V. van Kooten, Mike Horton, S. Wenninger, H. Babačić, B. Schoser, C. Lefeuvre, Najib Taouagh, P. Laforet, Sonia Segovia, Jordi Diaz-Manera, Kristl G. Claeys, Tiziana E Mongini, O. Musumeci, Antonio Toscano, T. Hundsberger, Esther Brusse, P. Van Doorn, A. T. van der Ploeg, N. A. van der Beek
0 28. 8. 2024.

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

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.


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