Patisiran: First Global Approval

@article{Hoy2018PatisiranFG,
  title={Patisiran: First Global Approval},
  author={Sheridan M. Hoy},
  journal={Drugs},
  year={2018},
  volume={78},
  pages={1625-1631}
}
Patisiran (ONPATTRO™) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. By specifically binding to a genetically conserved sequence in the 3′ untranslated region of mutant and wild-type transthyretin (TTR) messenger RNA, patisiran causes its degradation (via RNA interference) and subsequently a reduction in serum TTR protein levels and tissue TTR protein deposits. Patisiran has been developed by Alnylam Pharmaceuticals; it was recently… 
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