Effects of physical therapy on motor ability in patients with spinal muscular atrophy III: a study protocol for a...

Research investigating the efficacy of spinal muscular atrophy type II rehabilitation has yielded conflicting conclusions, underscoring the need for high-quality research to validate the role of physical therapy. Furthermore, exercise training should be considered as a potential non-pharmacological strategy to enhance motor function in SMA type III patients. Hence, this study is designed to explore the effectiveness of physical therapy for improving motor function in individuals with SMA type III.

This study is an open-label, randomised controlled trial. We will first stratify patients by disease severity (mild=independent walking, severe=non-ambulatory) and then randomly assign 428 participants to either the treatment group or the control group for a period of 12 weeks using a computer-generated randomisation schedule with a 1:1 allocation ratio. The intervention group will undergo a combination of cycling, resistance training, balance exercises, postural control training and locomotion exercises, while the control group will receive education sessions and a standard home exercise programme consisting of stretching and relaxation exercises. Outcome measurements will be assessed at baseline, immediately postintervention and at 3-month and 6-month follow-up assessments. The primary outcome will be assessed using the Hammersmith Functional Motor Scale Expanded, while secondary outcomes will include: Manual Muscle Test, Six-Minute Walk Test, 10 Metre Walk/Run Test, adverse events monitoring, Paediatric Quality of Life Inventory Multidimensional Fatigue Score and active and passive range of motion measurements.

The study protocol and consent form have been approved by the Ethics Committee on Biomedical Research of West China Hospital of Sichuan University (#2025-56) on 19 February 2025. Results will be published in peer-reviewed journals, presented at national and/or international conferences and disseminated to Chinese rare disease support groups.

ChiCTR2500101177.