NGLY1 and proximal spinal muscular atrophy: To date, there is one Food and Drug Administration (FDA)-approved AAV9 therapeutic, Onasemnogene Abeparvovec, for spinal muscular atrophy (SMA) treatment, and over 20 clinical trials involving the use of AAV9 in treating human Batten’s disease, Canavan disease, Duchenne Muscular Dystrophy, Pompe Disease, NGLY1 Deficiency, and muscle and heart diseases (see Table S1 with AAV9 clinical trials information).