DescriptionBranaplam Proposed Mechanism of Action.png
English: Branaplam acts to promote exon 7 inclusion in SMA patients by enhancing splicing at the exon 7-exon 8 junction. The small molecule (NVS-SM2) enhances recognition of SMN2 transcripts by U1-U2 specifically by enhancing the affinity of U1 to the 5'-splice site of SMN exon 7. Adapted from Palacino, J. et al. Nature Chemical Biology 2015, 11, 511.
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