Givosiran sodium is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran sodium downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran sodium can be used for the research of acute intermittent porpria.
References:
[1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339.
[2]. Balwani M, et, al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porpria. N Engl J Med. 2020 Jun 11;382(24):2289-2301.
















