RBM8A and amyotrophic lateral sclerosis: In ALS model mice overexpressing human Tar DNA–binding protein 43 (TDP-43), a decrease in ataxin-2 markedly improved survival and motor function by suppressing TDP-43 aggregation [40], and this led to the launch of a clinical trial of ATXN2-targeted antisense oligonucleotide treatment in ALS patients (ClinicalTrials.gov: NCT04494256).