Body Weight Support Harness System in Spinal Muscular Atrophy

Caregiver survey

A five question likert-style survey asking families to rate their experience during the study. The scales asks the family to rate (1) the perceived impact of BWSS on motor and (2) non-motor skills, (3) the child's perceived enjoyment using the system, (4) the ease of equipment use, and (5) if the family believed that use of the BWSS was beneficial on a 5-point Likert scale; scores of 4 or 5 were considered favorable, scores of 3 were considered neutral, and scores of 1 or 2 were considered unfavorable. Higher scores indicate greater perceived benefit of participation.

Time frame: End of study visit (month 6)

Change in The Neuromuscular Gross Motor Outcome (GRO) scale

A 50-item performance-based outcome developed to quantify motor function across the span of age and abilities in individuals with neuromuscular disorders including spinal muscular atrophy (SMA). Scores range from 0 - 100 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

Time frame: Completed at baseline, 3 months, and 6 months

Change in Hammersmith Functional Motor Scale Expanded (HFMSE)

A disease-specific and performance-based outcomes originally developed to measure motor skill in untreated individuals with SMA. Scores range from 0 - 66 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

Time frame: Completed at baseline, 3 months, and 6 months

Change in Revised Hammersmith Scale (RHS)

A disease-specific and performance-based outcomes originally developed to measure motor skill in untreated individuals with SMA, revised following Rasch analysis of HFMSE. Scores range from 0 - 69 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

Time frame: Completed at baseline, 3 months, and 6 months

Change in Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest

A norm-referenced developmental assessment of gross motor skill in children 16 days - 3.5 years of age. Raw scores range from 0 - 72. Higher scores indicate a greater level of funcion. An increase in score indicates a better outcome.

Time frame: Completed at baseline, 3 months, and 6 months

Change in World Health Organization Motor Milestone Checklist

A checklist of key gross motor milestones expected during typical development. Scores can range from 0 - 6 milestones achieved. Achievement of a greater number of milestones indicates better outcomes.

Time frame: Completed at baseline, 3 months, and 6 months