Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study.
Yuan M, Andrinopoulou ER, Kruijshaar ME, Lika A, Harlaar L, van der Ploeg AT, Rizopoulos D, van der Beek NAME
Orphanet journal of rare diseases, 2020 09 03
Abstract
Pompe disease is a rare, progressive metabolic myopathy. The aim of this study is to investigate the associations of physical outcomes with patient-reported outcome measures (PROMs) in late-onset Pompe disease.