Lowering ATXN2 expression with antisense oligonucleotide (ASO) therapy and genetic interaction proved to be efficient in preclinical models of ALS and SCA2 (12, 18), and phase 1 trials are currently underway in ALS patients for the ATXN2 ASO therapeutic BIIB105. This evidence concerns the gene ATXN2 and amyotrophic lateral sclerosis.