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Article

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

10 Jun 2020

Abstract excerpt

BACKGROUND: Up-regulation of hepatic delta-aminolevulinic acid synthase 1 (ALAS1), with resultant accumulation of delta-aminolevulinic acid (ALA) and porphobilinogen, is central to the pathogenesis of acute attacks and chronic symptoms in acute hepatic porphyria. Givosiran, an RNA interference therapy, inhibits ALAS1 expression. METHODS: In this double-blind, placebo-controlled, phase 3 trial, we randomly...

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PMID: 32521132
DOI: 10.1056/nejmoa1913147

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