Patisiran: First Global Approval
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DOI: 10.1007/s40265-018-0983-6
Abstract: 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… read more here.
Keywords: patisiran; ttr; global approval; patisiran first ... See more keywords