Tofersen, an antisense oligonucleotide therapy which targets SOD1 mRNA (Tzeplaeff et al., 2023), is restricted to specific familial cases of ALS that make up only a very small proportion of ALS cases (Tzeplaeff et al., 2023), leaving the large majority of patients without effective therapies. This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.