SMN1 and spinal muscular atrophy, type 1: Indeed, the delivery of the SMN1 gene (SMN cDNA) to infants with SMA type 1 using adeno-associated virus (AAV) serotype 9 dramatically improved neuromuscular function and prolonged life expectancy, making the case for approval of the drug, named Translarna (onasemnogene abeparvovec), by North American and European regulatory agencies [68].