Nusinesen is used to treat spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease caused by homozygous loss or mutation of the survival motor neuron 1 (SMN1) gene (Chiriboga, 2017; Crooke et al., 2018; Li, 2020). This evidence concerns the gene SMN1 and proximal spinal muscular atrophy.