SMN2 and proximal spinal muscular atrophy: Over the past years, novel treatments, such as the SMN2 splicing modifiers Nusinersen [3, 4] and Risdiplam ([5, 6]; ClinicalTrials.gov: NCT02913482, NCT02908685), as well as Onasemnogene Abeparvovec [7], as first gene replacement therapy, have been developed for SMA.