The intrathecally delivered antisense oligonucleotide, nusinersen (Spinraza®) (Darras et al, 2019; Finkel et al, 2017; Hua et al, 2007; Hua et al, 2008; Ramos et al, 2019; Singh et al, 2009) and the oral small molecule, risdiplam (Evrysdi®) (Darras et al, 2021; Gowda et al, 2022; Singh et al, 2020), are splicing modulators that increase the amount of SMN2-derived transcripts that retain exon 7 and are approved for pediatric and adult SMA patients. This evidence concerns the gene SMN2 and proximal spinal muscular atrophy.