SMN2 and spinal muscular atrophy: These include Patisiran (Onpattro) and Vutrisiran (Amvuttra), both siRNAs for treating hereditary transthyretin-mediated amyloidosis; Nusinersen (Spinraza), an ASO that modifies SMN2 splicing; and Risdiplam (Evrysdi), a small molecule that promotes exon 7 inclusion, both used in spinal muscular atrophy.