SMN2 and proximal spinal muscular atrophy: Based on the mode of discovery of the DAQ-DcpSi in a screen utilizing an SMN2 promoter-driven reporter assay and reported effects on SMN2 transcripts, SMN protein levels and nuclear gem counts, this class of compound have been assumed to exert their therapeutic effects in SMA mice via elevation of SMN consequent to SMN2 promoter induction.