All of these studies indicate that, while motor neuron death itself can be rescued by targeting the pathways that directly lead to motor neuron death (such as p53 activation, and Mdm2 and Mdm4 missplicing), this does not rescue motor neuron dysfunction or other motor neuron-associated phenotypes in models of SMA (Lotti et al., 2012; Van Alstyne et al., 2018). The gene discussed is MDM4; the disease is proximal spinal muscular atrophy.