Stander Use in Spinal Muscular Atrophy: Results From a Large Natural History Database

Purpose: The purpose of this study was to describe stander use in a natural history cohort of drug therapy-naive children with spinal muscular atrophy (SMA) who are not walking and identify factors associated with consistent stander use. Methods: Data from 397 children with SMA types 1 and 2 characterized the prevalence and frequency of stander use. Predictors of consistent stander use explored were SMA type, survival motor neuron 2 gene (SMN2) copy number, respiratory support, and motor performance. Results: Prevalence of consistent stander use was 13% in type 1 and 68% in type 2. SMA type,SMN2copy number, respiratory support, and head rotation control each predicted consistent stander use. Conclusions: Findings characterize stander use in children with SMA who are not walking, address important safety considerations, identify factors that may inform physical therapists' clinical decision-making related to standing program prescription, and provide guidance for future prospective studies.