Mutations in VCP have been connected to neurodegenerative multisystemic proteinopathies in humans, such as inclusion body myopathy with Paget disease of bone and frontotemporal dementia (IBMPBFD) (Kimonis et al. 2000), amyotrophic lateral sclerosis (ALS) (Johnson et al. 2010), and Charcot-Marie-Tooth disease type 2 (CMT2) (Gonzalez et al. 2014). Here, VCP is linked to amyotrophic lateral sclerosis.