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givosiran

Ligand Summary
Description
Givosiran is a double-stranded small interfering RNA that causes degradation of aminolevulinate synthase1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP.
Synonyms & Links
DrugCentral: 5354
LyCHI:  givosiran


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