These data clearly show that the SOD1-Derlin-1 interaction inhibitor can ameliorate ALS pathology both in in vitro human model and in vivo mouse model, demonstrating the importance of the SOD1-Derlin-1 interaction in the pathogenesis of SOD1mut-induced FALS and the potential of the SOD1-Derlin-1 interaction as a therapeutic target in ALS. Here, DERL1 is linked to amyotrophic lateral sclerosis.