HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness

This pilot study is evaluating a 90-hour intensive motor training program — called HABIT-ILE combined with functional strength training (FST) — delivered as a 3-week summer camp for children with spinal muscular atrophy (SMA) who are already on disease-modifying drug therapies. SMA is a genetic condition causing muscle weakness, and while newer drugs have transformed outcomes, rehabilitation remains essential, and this type of intensive training has shown benefits in children with cerebral palsy. Children aged 5 to 17 with a confirmed SMA diagnosis, who can stand with support (SMA type 2 or 3), are on a stable drug therapy dose for at least 6 months, and speak English are eligible, while those with recent surgery or participation in another trial are excluded. Participants will attend a 6-hour-per-day camp for three weeks involving intensive task-specific motor exercises and strength activities, with follow-up assessments at 6 months. This summary was prepared to help patients understand the study in plain language.