Explore the utility of an in-home, multi-directional, body-weight support harness system (BWSS) to maximize the therapeutic benefit of novel therapies for the treatment of spinal muscular atrophy (SMA).

SMA is a disease involving the degeneration of lower motor neurons in the spinal cord leading to progressive muscle atrophy. The recent success of novel therapies has changed the natural course of the disease by prolonging the lifespan and allowing the attainment of motor milestones never before seen in SMA. However, early deficits in muscle strength limit mobility and individuals with SMA still do not experience the typical developmental path, putting them at a disadvantage for learning more advanced skills. Additionally, they are typically older and larger before they start attempting weight-bearing activities such as standing and walking and the ability to practice these challenging movements is limited. The BWSS allows children to spend extended periods playing in an upright position to more closely mimic typical development.

We describe an interventional study in which BWSS were set-up in the family’s home and caregivers were educated on its use. Caregivers were asked to engage in play activities with the child using BWSS for two hours a day over three months and document the frequency of use. Evaluation of gross motor skills was completed at baseline and three-months using the Bayley Scales of Infant and Toddler Development, the Hammersmith Functional Motor Scale-expanded, the World Health Organization Motor Milestones, and the Neuromuscular GRO.

Preliminary results suggest improvements in motor outcomes. Up to date functional outcome data will be presented at time of meeting.

Daily in-home use of a multi-directional BWSS for up to two hours each day can provide children with the opportunity to practice building strength in standing and walking to make a meaningful change compared to traditional therapy.