Testing HABIT-ILE and Functional Strength Training for Children Ages 5 to 17 with Spinal Muscular Atrophy SMA Receiving Disease-Modifying Therapies

What this Trial Is About

This research aims to evaluate the preliminary effectiveness of an intensive motor skill intervention combining Hand-Arm Bimanual Intensive Therapy Including Lower Extremities HABIT-ILE with functional strength training FST in children aged 5 to 17 years with Spinal Muscular Atrophy SMA who are also receiving disease-modifying therapies DMTs. SMA is a serious neuromuscular condition causing muscle weakness and atrophy, and despite advances in drug treatments, rehabilitation remains essential to improve motor function. The study focuses on how combining HABIT-ILE and FST may enhance motor skills and functional independence in this population. Participants will take part in a combined HABIT-ILE and FST program delivered through a summer camp format, engaging in 6 hours of training per day for 3 weeks, totaling 90 hours. This program integrates goal-directed, task-specific training for both upper and lower limbs with strength exercises tailored to individual goals. The intervention emphasizes motor learning principles, postural control, and increasing endurance and strength, aiming to complement their ongoing DMTs. During the study, children will complete assessments at baseline, immediately after the intervention, and at 3 months follow-up, including the Hammersmith Functional Motor Scale - Expanded HFMSE to measure motor function. Additional tests such as surface electromyography sEMG, endurance, walking, and functional mobility assessments will also be conducted. Daily activity logs and a feasibility questionnaire will track adherence and participant experience. The total involvement spans at least 3 months, with researchers monitoring motor skills, muscle fatigability, and functional independence over time.

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