As the first approved small molecule splicing modifier drug, Risdiplam (Evrysdi) is granted approval by the European Commission to expand the EU marketing authorization. This extension now includes infants with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months. Type 1 spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease and a leading genetic factor of infant mortality, that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam modifies SMN2 pre-messenger RNA splicing and increases levels of functional SMN protein in both the central nervous system and peripheral organs.