ATXN2 and amyotrophic lateral sclerosis: Efforts are underway to test the efficacy of this drug in ameliorating ALS phenotypes in other mouse models of ALS with later disease onset (Arnold et al., 2013); we remain encouraged by the ability of etidronate to lower ataxin-2 levels in vivo, as ATXN2 is a previously validated therapeutic target in mouse models of ALS (Becker et al., 2017) and SCA2 (Scoles et al., 2017) as well as by human genetics (Elden et al., 2010; Scoles and Pulst, 2018).