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1809 Givosiran
a05ba268e393fe6c6d947eaf5d6c97ef4d5993c5dbba029513246f414a58053b

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Properties

Chemistry

Givosiran, sold under the brand name Givlaari, is for the treatment of adults with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood). Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, an important enzyme in the production of heme.

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