Feasibility and Preliminary Effectiveness of HABIT-ILE + Functional Strength Training in Children With Spinal Muscular Atrophy (SMA): a Prospective Single-group Intervention Trial.
Teachers College, Columbia University
Summary
This single-arm pilot study will assess the preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying therapies. Participants will attend a HABIT-ILE + FST summer camp for 6 hours per day over a 3-week period, totaling 90 hours of training.
Description
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder marked by the progressive degeneration of alpha motor neurons in the spinal cord and brainstem, resulting in proximal muscle atrophy and weakness. Based on age of onset and the motor function achieved, children were historically categorized into functional groups. However, recent advances leading to the development of disease-modifying therapies (DMTs) have transformed the management of SMA. Outcomes are now primarily determined by the timing of DMT initiation, with early treatment-ideally before symptom onset-showing significant…
Eligibility
- Age range
- 5–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Have a confirmed SMA diagnosis * Be 5-17 years old * Complete all pre- and post-intervention assessments * Understand and speak English * Follow instructions without major cognitive or behavioral issues * Have received a stable disease-modifying therapy (DMT) dose for ≥6 months * Be classified as a sitter or walker (SMA type 2 or 3) with supported standing ability (score ≥1 on Item 18 of the HFMSE) without KAFOs, AFOs, or external standing devices Exclusion Criteria: * Orthopedic surgery within the past year * New pharmaceutical treatment during the study * Concurrent…
Interventions
- OtherCombined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
Goal-directed, task-specific training for both the upper and lower extremity, and postural control with targeted strengthening exercises to enhance motor control and facilitate the achievement of functional goals
Location
- Center for Cerebral Palsy Research, Teachers College, Columbia UniversityNew York, New York