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This review identifies key clinical outcome assessments for measuring treatment benefits in Pompe disease, suggesting improvements.
Several COIs were identified from the literature that may be of importance to patients with Pompe disease. Individual COAs frequently used in assessing these concepts were found to have gaps with regards to content validity and psychometric properties. Additional research with patients with Pompe disease could be considered to address issues of content validity. Furthermore, the use of several COAs could be considered in future studies to capture what matters most to patients with Pompe disease.
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Rofail et al. (2026) studied this question.
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