Grant: Doctoral Award - Frederick Banting and Charles Best Canada Graduate Scholarships

Spinal muscular atrophy (SMA) is the leading genetic cause of infant deaths affecting 1 in 10,000 live births. SMA is characterized by the degeneration of motor neurons and skeletal muscle atrophy. This leads to proximal limb and trunk muscle wasting that progress to paralysis and ultimately death in severe cases. In the present proposal, I will exploit skeletal muscle as a therapeutic target to treat SMA. Exercise therapy is proven to benefit skeletal muscle, motor neurons and the neuromuscular junction. I hypothesize that exercise alone or in combination with exercise mimetics or muscle growth factors will be of benefit in SMA. I will investigate whether exercise and exercise mimetics can lead to wide-spread benefits in a novel mouse model of intermediate SMA. Based on several observations related to the motor neuron diseases amyotrophic lateral sclerosis and spinal and bulbar muscular atrophy, I will use a potent muscle regulator together with exercise to rescue SMA mice. The results of this study will significantly advance SMA therapeutic research and hold great promise in treating patients suffering from SMA.