While the mutations associated with both the sporadic and familial forms of the disease can be varied, aggregates of hyperphosphorylated, ubiquitinated and truncated TAR DNA-binding protein 43 (TDP-43) within patients’ motor neurons and glial cells is a common feature in 97% of ALS cases [1,2,3]. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.