Body Weight Support Harness System in Spinal Muscular Atrophy

Exploring the Feasibility and Utility of In-home Body Weight Support Harness System Use in Children Treated for Spinal Muscular Atrophy: a Pilot Study

The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are:

1. Is the in-home body weight support harness system a feasible option for families to use?
2. Is the in-home body weight support harness system a useful tool for children treated for spinal muscular atrophy?
3. Is the in-home body weight support harness system a safe tool for children treated for spinal muscular atrophy?

Participants will be given an in-home body weight support harness system and taught how to use it. Families will document how often and for how long they use the system over 6 months. Children will be given tests of motor function at the beginning, 3-months, and 6-months. At the end of the study, families will be asked to fill out a questionnaire about thier experience using the system.

Inclusion Criteria:

• Confirmed heterozygous mutation in SMN1 gene and 2 or 3 copies of SMN2 (historically, SMA Type 1 and Type 2)
• Past or current treatment with nusinersen, onasemnogene abeparvovec, or risdiplam
• Upright head control (defined as the ability to lift head from full forward flexion)
• Weight under or 50lbs
• Confirmed motor delay

Exclusion Criteria:

• 4+ copies of SMN2 (historically, SMA Type 3 and 4)
• Evidence of lower limb injury or recent fracture
• In the opinion of the investigator, it was unsafe for the child to participate