We previously reported that a 2′-O-(2-methoxyethyl) (MOE) therapeutic ASO (ASO-10-27 or ISIS-SMNRx) that promotes exon 7 inclusion in SMN2, rescues the phenotypes of several SMA mouse strains (Hua et al, 2010, 2011; Passini et al, 2011; Sahashi et al, 2012). This evidence concerns the gene SMN2 and proximal spinal muscular atrophy.