Identification of ALS-causative genes, including superoxide dismutase 1 (SOD1), transactive response DNA-binding protein 43 (TARDBP-43), fused in sarcoma (FUS), chromosome 9 open reading frame 72 (C9orf72), heterogeneous nuclear ribonucleoprotein A1 (HNRNPA1), valosin-containing protein (VCP), ubiquilin 2 (UBQLN2), sequestosome 1 (SQSTM1/p62), annexin A11 (ANXA11), optineurin (OPTN), and TANK (TRAF-associated NF-κB activator)-binding kinase 1 (TBK1) have advanced the understanding of ALS pathogenesis. The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.