SMN1 and spinal muscular atrophy: Interestingly, defective WRAP53β-mediated trafficking of SMN is observed in patients afflicted by the most severe form of spinal muscular atrophy (type I or Werdnig-Hoffmann disease) (Lefebvre et al., 1997; Oskoui et al., 2007; Mahmoudi et al., 2010; Tapia et al., 2012).