NINDS' research goal for Spinal Muscular Atrophy is to efficiently translate a mechanistic understanding of SMA into a testable therapy suitable for clinical trials. The NINDS strategy for achieving its research goal is to fund parallel therapeutic pathways in a rapid milestone-driven process that will identify a promising therapeutic candidate. This project will require a high level of scientific expertise and experience on the part of the contractor. Furthermore, efficient progress will involve an unusual degree of acceleration and integration of multiple research projects, and thus success will also require that the contractor use creativity and flexibility in applying that scientific expertise.