In April 2023, the SOD1-targeting ASO drug Tofersen was approved by the U.S. Food and Drug Administration (FDA) after promising clinical trials in ALS patients with a mutation in the SOD1 gene [435], providing a first proof-of-concept that this is a viable approach to reduce aggregation of pathogenic proteins in ALS. This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.