The roadmap for ASOs being clinically impactful has been realised for several neurological disorders including spinal muscular atrophy (using a splice switching MIXmer ASO to promote exon 7 inclusion in SMN2 and thereby increase functional SMN expression) and SOD1-related amyotrophic lateral sclerosis (using a GAPmer ASO to reduce SOD1 levels). This evidence concerns the gene SMN2 and proximal spinal muscular atrophy.