SMN2 and proximal spinal muscular atrophy: Interestingly, neuron-iPSC from SMA patients were also used to test the efficacy of the recent FDA approved small molecule EvrysdiTM (risdiplam) (Ratni et al., 2016; Ratni et al., 2018; Dhillon, 2020), which forces the inclusion of exon 7 and thus restore SMN protein levels (Poirier et al., 2018).