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). The gene discussed is SOD1; the disease is nervous system disorder.