C3 and spinal muscular atrophy, type 1: We performed transcriptome-wide sequencing of RNA from type 1 SMA patient fibroblasts treated with SMN-C3 at ~50 times the EC50 (500 nM, Supplementary Fig. 6A) or NVS-SM1 at ~5 times the EC50 (24 nM, Supplementary Fig. 6B).