SMN2 and proximal spinal muscular atrophy: published the first report of a phenotypic screen yielding selective SMN2 splice modulators, leading to Risdiplam (RG7916), a recently FDA-approved drug for SMA treatment (Naryshkin et al., 2014; FDA Approves Genentech’s Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older, 2020), while in 2015, Palacino’s group identified the splicing modulator Branaplam (LMI070) (see section Direct and Indirect Modulation of Survival Motor Neuron 2 Transcription) (Palacino et al., 2015).