TAR DNA-binding protein 43 (TDP-43) is the major pathological inclusion protein involved in the pathogenesis of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to TDP-43 pathology (FTLD-TDP), providing a molecular link that put these disorders in a common ALS-FTLD disease spectrum [1–3]. The gene discussed is PROS1; the disease is amyotrophic lateral sclerosis.