ASO treatment has found increased application in clinical development in ALS; initially by targeting of the SOD1 gene for ALS patients harboring an ALS-causing mutation in this gene in clinical trials [88], and with the additional development of ASO treatment for C9orf72 repeat expansion carriers (NCT03626012, NCT04288856) as well as the targeting of the CAG repeat expansion of the genetic disease modifier ATXN2 (NCT04494256). This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.