Given the recent hypothesis on the role of RNA and splicing abnormalities as a primary determinant of selective MN death in SMA [52,53], the experiment was then directed to the analysis of transcriptional changes in untreated SMA iPSC-derived MNs compared to heterozygous iPSC- and treated SMA iPSC-MNs. Here, SMN1 is linked to proximal spinal muscular atrophy.