Hereditary Inclusion Body Myopathy, Paget Disease of Bone, Frontotemporal Dementia (IBMPFD) and Amyotrophic Lateral Sclerosis (ALS), recently termed VCP-associated disease (VCP disease) is a multisystem disorder with a diverse collection of manifestations caused by mutations in the valosin containing protein (VCP) gene [1–4]. The gene discussed is VCP; the disease is amyotrophic lateral sclerosis.