VCP and amyotrophic lateral sclerosis: One known heterozygous missense mutation in the VCP gene, c.463C>T (p.R155C) (Figure 1A) was identified in one familial ALS proband (III-6) and his affected sister (III-5) (Figure 1B), as well as another sporadic patient in the cohort of 275 sporadic ALS cases and 15 familial ALS pedigrees.