Abstract
Objectives: Acute Hepatic Porphyria (AHP) is a family of rare genetic diseases leading to an enzyme deficiency in the heme biosynthesis pathway, causing accumulation of neurotoxic heme intermediates, resulting in neurovisceral attacks and chronic manifestations. Givosiran, an investigational RNAi therapeutic, is being evaluated for its ability to reduce the levels of neurotoxic intermediates thus decreasing attacks and disease manifestations.
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