Body Weight Support Harness System in Spinal Muscular Atrophy
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
About this trial
This is an interventional treatment trial for Spinal Muscular Atrophy Type I focused on measuring exercise, physical therapy
Eligibility Criteria
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
Sites / Locations
- Nationwide Children's Hospital
Arms of the Study
Arm 1
Experimental
In-home body weight support harness system
All participants will be entered into treatment arm and receive an in-home body weight support harness system