Importantly, pharmacological inhibition of β-catenin dramatically ameliorated neuromuscular pathology in zebrafish, Drosophila and mouse models of SMA (Wishart et al., 2014) while systemic Uba1 gene therapy increased survival and improved neuromuscular and peripheral pathology of SMA mice (Powis et al., 2016). Here, UBA1 is linked to proximal spinal muscular atrophy.