As discussed above, postnatal treatment of NS-ML mouse models with inhibitors of the PI3K/mTOR/AKT pathway or with the tyrosine kinase inhibitor dasatinib reversed cardiomyopathy phenotypes (Marin et al., 2011; Wang et al., 2017; Yi et al., 2016), and treatment of a NS-ML patient with the mTOR inhibitor everolimus provided clinical benefit (Hahn et al., 2015). Here, MTOR is linked to cardiomyopathy.