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

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.