In ALS, skeletal muscle harbors the characteristic protein aggregates (composed principally of TDP-43) (Cykowski et al., 2018) whose burden in the central nervous system (CNS) predicts neurodegeneration (Brettschneider et al., 2014; Mackenzie and Neumann, 2016). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.