Grant: Operating Grant: Clinical Trials Projects

Spinal muscular atrophy (SMA) is a rare disease that weakens muscles, limits independent physical function, and shortens children's lives. In the last 5 years, new drug therapies have helped children with SMA live longer and get stronger. However, we don't know how best to give these children fun, convenient opportunities to participate in physical activities that help them undertake everyday activities that matter to them. We also need better ways to measure if what we're doing is helping children in a meaningful way. One promising option is using active video exergames, which are computer games designed to make exercise fun, motivating and convenient to undertake at home. In partnership with children with SMA, their therapists, and their doctors, we co-developed an exergame that uses a low-cost sensor to track body movements and measure progress. Seventy-four children with SMA (ages 6-18 years) will participate in either the exergaming treatment for 12 weeks or continue with their usual care where they will continue to benefit from physical activities in their community. All children will wear a small device on their wrist to see how well it measures changes in children's arm movements during their daily activities at home. We will compare children's' and families' perception and satisfaction of their performance in important activities of daily living to evaluate the impact of the exergaming intervention. We will also test children's motor abilities before and after the intervention. Our study results will help us understand whether exergaming is a good way to provide accessible rehabilitation that promotes physical activity and independent participation in activities of daily living for children with SMA. We will also understand how to best measure the impact of our treatments in a meaningful way. Ultimately, we can use this information to make informed care decisions to optimize children's individual outcomes.