Description
A SMN2 splicing modifier; enhances SMN2 splicing (EC1.5x= 40 nM) and increases SMN protein levels (EC1.5x = 163 nM) in cellular assays; increases brain and quadricep muscle SMN protein levels in the adult C/C-allele mouse model of SMA and the SMND7 neonate mouse model of severe SMA at 3 mg/kg; increases vGLUT1 proprioceptive inputs on motor neurons, the number of L3-L5 motor neurons, the percentage of fully innervated NMJs, and the size of the extensor digitorum longus muscles in adult SMND7 mice