The impact of early powered mobility training in families and children with Spinal Muscular Atrophy Type I.

Abstract

This study examined the effects of early powered mobility training using modified ride-on toy cars in young children with Spinal Muscular Atrophy type I (SMA I). A randomized, waitlist-controlled, single-blinded trial was conducted in Spain with 16 children aged 10 months to 5 years. The intervention consisted of 12 weeks of powered mobility training in natural environments, followed by 4 weeks of follow-up. Primary outcomes included participation (YC-PEM), functional ability (PEDI-CAT), and quality of life (PedsQL-NM), assessed at five time points. Parental stress (PSI-SF) was a secondary outcome. Children in the experimental group demonstrated clinically meaningful gains in Daily Activities and Mobility from week 4 onward (z = 0.41-0.72; g = 0.29-0.53), while Social/Cognitive improvements exceeded SEMm by weeks 8-16. Quality of life showed significant differences at week 4 (p = 0.03; g = 0.93), though below the SEMm threshold. Modest increases were observed in home participation, while Desired Change remained stable. Parental stress reductions reached statistical significance at weeks 4, 8, and 16 (p < 0.05), though within SEM_m margins. Findings support early powered mobility as an assistive technology that enhances functional independence in SMA I. Complementary psychosocial and environmental supports may maximize broader participation and quality-of-life outcomes.