Among these potentially targetable NDDs and variants are AON strategies that show promising results in preclinical studies or tentative success in clinical trials including the TANGO strategy for Dravet syndrome and SYNGAP1 syndrome (OMIM: 612621), exon skipping for reading frame restauration for DMD and AON-mediated degradation of lncRNAs for Angelman syndrome and CHD2 syndrome (OMIM: 615369). The gene discussed is DMD; the disease is encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.