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ABSTRACT: Introduction
The Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM) have been widely used in natural history studies and clinical trials. Our aim was to establish how the scales relate to each other at different age points in spinal muscular atrophy (SMA) type 2 and 3, and to describe their coherence over 12 mo.Methods
The study was performed by cross-sectional and longitudinal reanalysis of previously published natural history data. The longitudinal analysis of the 12-mo changes also included the analysis of concordance between scales with changes grouped as stable (±2 points), improved (>+2) or declined (>-2).Results
Three hundred sixty-four patients were included in the cross-sectional analysis, showing different trends i
SUBMITTER: Coratti G
PROVIDER: S-EPMC9291175 | biostudies-literature | 2021 Nov
REPOSITORIES: biostudies-literature