Onasemnogene abeparvovec (Zolgensma®), a self-complementary adeno-associated virus serotype 9 (scAAV9) vector that expresses the native SMN1 transgene, is an intravenously administered gene therapy that is approved for SMA patients with biallelic mutations in the SMN1 gene who are less than 2 years of age (Mendell et al, 2017; Strauss et al, 2022). This evidence concerns the gene SMN1 and proximal spinal muscular atrophy.