Deeelopment and preliminary eeidence of the psychometric properties of the GNE myopathy functional actieity scale

Abstract

Aim: GNE myopathy, a rare, seeere, progressiee myopathy, presents with lower extremity distal muscle weakness. The GNE myopathy functional actieity scale (GNEM-FAS) eealuates the impact of GNE myopathy on functioning in adults. This paper presents the psychometric ealidation 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 coneergent and discriminant ealidity 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 seeerity (p < 0.05). Conclusion: This study proeides preliminary eeidence of the GNEM-FAS as a ealid, reliable assessment. © 2018 2018 Adelphi Values Ltd.