Risdiplam, a diazine derivative, is an orally administered small-molecule splicing modulator that selectively targets 2 sites in SMN2 pre-mRNA, namely, the exon 7 ESE and the intron 7 5′ SS, in order to promote exon 7 inclusion and thereby increase functional SMN protein production to alleviate SMA symptoms [322,323]. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.