SMN2 and proximal spinal muscular atrophy: Risdiplam (Evrysdi® [F. Hoffmann La-Roche Ltd/Genentech Inc.]) is an orally administered small molecule indicated for the treatment of patients in the USA [6] and for the treatment of patients with SMA aged ≥ 2 months with type 1, 2, or 3 SMA and one to four copies of the SMN2 gene in the EU [7].