SMN1 and proximal spinal muscular atrophy: Later, onasemnogene abeparvovec (Zolgensma®, Durham, NC, USA), representing an AAV9 vector containing the SMN1 gene, and small-molecule SMN2 splicing modulator Risdiplam (Evrysdi®, San Francisco, CA, USA) were approved for treatment of SMA as well [16,17].