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Article

Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

12 Oct 2021

Abstract excerpt

BACKGROUND: Late-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients' daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform...

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PMID: 34641935
DOI: 10.1186/s13023-021-02067-x

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