FightSMA (Spinal Muscular Atrophy) Grants

FightSMA is dedicated to supporting the development of a treatment for SMA.
Basic research supported by FightSMA focuses upon furthering the understanding of the pathogenesis of SMA. This includes (but is not limited to) identifying SMN's function in neurons; why a motor neuron depleted of SMN has a limited life span; what distinguishes a motor neuron which survives from one that dies; whether SMA is purely a motor neuron autonomous disorder; the temporal/development window in which low SMN confers motor neuron vulnerability and conversely the window in an organism with reduced SMN levels when SMN repletion has a therapeutically significant impact; understanding the mechanisms of SMN gene regulation; identifying molecular targets for potential therapies.
Translational research includes the development of reagents that will aid in the identification of potential treatments. For example, the development of a screening procedure or cellular (e.g. organotypic cultures) and animal (e.g. severe, intermediate, mild SMA mice) model that would support the SMA community’s goal of identifying a treatment. Translational research also includes the identification of individual and classes of non-SMN2 inducing agents which are the most neuroprotective in SMN depleted states.
Fast-Track Status: A one page pre-proposal requesting “fast track” status will be reviewed and a decision made by the Research Advisory Board. Applications accepted for fast track status will be reviewed upon receipt of the application.