Small molecules targeting DCPS have been employed in early trials for spinal muscular atrophy (SMA) (Gogliotti et al., 2013), and more recent development of proteolysis targeting chimeras (PROTACs) targeting DCPS (Swartzel et al., 2022) may hold future promise as a therapeutic in AML. The gene discussed is DCPS; the disease is proximal spinal muscular atrophy.