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Article

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

6 Feb 2019

Abstract excerpt

BACKGROUND: Induction of delta aminolevulinic acid synthase 1 ( ALAS1) gene expression and accumulation of neurotoxic intermediates result in neurovisceral attacks and disease manifestations in patients with acute intermittent porphyria, a rare inherited disease of heme biosynthesis. Givosiran is an investigational RNA interference therapeutic agent that inhibits hepatic ALAS1 synthesis. METHODS: We conducted a...

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PMID: 30726693
DOI: 10.1056/nejmoa1807838

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