Abstract:
AIM:GNE myopathy, a rare, severe, progressive myopathy, presents with lower extremity distal muscle weakness. The GNE myopathy functional activity scale (GNEM-FAS) evaluates the impact of GNE myopathy on functioning in adults. This paper presents the psychometric validation of the GNEM-FAS. PATIENTS & METHODS:Validation of the GNEM-FAS was performed using data from a randomized, double-blind, placebo-controlled Phase-II study (n = 46). RESULTS:Domain score distributions were acceptable. Moderate inter-item correlations (typical range, 0.40-0.70), strong item convergent and discriminant validity and high internal consistency reliability (α = 0.88-0.92) supported the instrument structure. Test-retest reliability was strong (ICC range: 0.87-0.95). Scale scores distinguished among subjects with differing disease severity (p < 0.05). CONCLUSION:This study provides preliminary evidence of the GNEM-FAS as a valid, reliable assessment.
journal_name
J Comp Eff Resjournal_title
Journal of comparative effectiveness researchauthors
Mayhew J,Bonner N,Arbuckle R,Turnbull A,Bowden A,Skrinar Adoi
10.2217/cer-2017-0062subject
Has Abstractpub_date
2018-04-01 00:00:00pages
381-395issue
4eissn
2042-6305issn
2042-6313journal_volume
7pub_type
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
pub_type: 杂志文章,评审
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journal_title:Journal of comparative effectiveness research
pub_type: 杂志文章
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
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journal_title:Journal of comparative effectiveness research
pub_type: 杂志文章
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journal_title:Journal of comparative effectiveness research
pub_type: 杂志文章
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journal_title:Journal of comparative effectiveness research
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