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familial dysautonomia

" in MedChemExpress (MCE) Product Catalog:
Cat. No. 상품명 Target 연구분야 Chemical Structure
  • HY-147149

    DNA/RNA Synthesis Neurological Disease
    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) (EC50 = 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 .
    BPN-15477

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