Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

Abstract Background The construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) questionnaire were evaluated for patients with late-onset Pompe disease (LOPD), a rare, autosomal recessive, progressive neu...

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Bibliographic Details

Published in:	Journal of Patient-Reported Outcomes
Main Authors:	Priya S. Kishnani, Simon Shohet, Syed Raza, Noemi Hummel, Jeffrey P. Castelli, Sheela Sitaraman Das, Heng Jiang, Agnieszka Kopiec, Ian Keyzor, Andreas Hahn
Format:	Article
Language:	English
Published:	SpringerOpen 2024-01-01
Subjects:	Patient-reported outcomes Patient-Reported Outcome Measurement Information System (PROMIS) Late-onset Pompe disease Physical function Quality of life PROPEL
Online Access:	https://doi.org/10.1186/s41687-024-00686-z