SOD1 and amyotrophic lateral sclerosis: In April 2023, a novel antisense oligonucleotide (ASO)-based therapy called tofersen received accelerated approval from the U.S. Food and Drug Administration (FDA) for treating adults with amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene which accounts for about 20% of familial ALS cases and up to 2% of sporadic cases (Figure 2) [102].