Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.
Harmke A van KootenMike C HortonStephan WenningerHaris BabačićBenedikt SchoserClaire LefeuvreNajib TaouaghPascal LaforêtSonia SegoviaJordi Díaz-ManeraKristl G ClaeysTiziana MonginiOlimpia MusumeciAntonio ToscanoThomas HundsbergerEsther BrussePieter A van DoornAns T van der PloegNadine A M E van der Beeknull nullPublished in: European journal of neurology (2024)
After removing one item, the modified-R-PAct scale is a valid disease-specific patient-reported outcome measure for patients with Pompe disease across multiple countries.