Accumulating evidence highlights the link between VCP mutations and multisystem proteinopathy, a spectrum of phenotypes which includes myopathy with rimmed vacuoles or inclusion body myopathy (IBM), early-onset Paget disease of the bone (PDB), as well as FTD and ALS (Kovach et al., 2001; Watts et al., 2004, 2007; Johnson et al., 2010; Abramzon et al., 2012; Benatar et al., 2013). Here, VCP is linked to amyotrophic lateral sclerosis.