Ongoing clinical trials, such as BIIB078 (NCT03626012), an investigational ASO for C9orf72-associated ALS that degrades G4C2 expansion-containing mRNA, and WVE-004 (NCT04931862), an ASO targeting repeat-containing pre-mRNA variants in patients with c9ALS/FTD, aim to evaluate these effects. This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.