BPN-15477 is an orally active, blood-brain barrier-permeable splicing modulator compound ( SMC ). BPN-15477 restores the correct splicing of exon 20 of the elongator complex protein 1 ( ELP1 ) ( EC 50 = 1.9 μM), thereby significantly increasing the in vivo functional protein levels in all tissues including the brain. BPN-15477 can be used in the research of familial dysautonomia, such as frontotemporal dementia.
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