An outstanding example are the variants of SOD1 which are targeted by Tofersen, an antisense oligonucleotide (Miller et al., 2022) Tofersen, a treatment designed specifically for SOD1-associated ALS was approved in April 2023 by the FDA after clinical trials (Miller et al., 2020, 2022; Benatar et al., 2022). This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.