EXPLORE B: A prospective, long‐term natural history study of patients with acute hepatic porphyria with chronic symptoms
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DOI: 10.1002/jimd.12551
Abstract: One‐year data from EXPLORE Part A showed high disease burden and impaired quality of life (QOL) in patients with acute hepatic porphyria (AHP) with recurrent attacks. We report baseline data of patients who enrolled in… read more here.
Keywords: long term; acute hepatic; disease; chronic symptoms ... See more keywords
Givosiran for the treatment of acute hepatic porphyria
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DOI: 10.1080/17512433.2022.2075848
Abstract: ABSTRACT Introduction Acute hepatic porphyrias (AHPs) are a family of rare inherited disorders characterized by enzyme dysfunctions in the hepatic pathway of heme biosynthesis. In AHPs, accumulation of the neurotoxic porphyrin precursors delta-aminolevulinic acid and… read more here.
Keywords: givosiran; acute hepatic; hepatic porphyria; givosiran treatment ... See more keywords
Acute hepatic porphyria and maternal health: Clinical and biochemical follow-up of 44 pregnancies.
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DOI: 10.1111/joim.13376
Abstract: BACKGROUND Pregnancy in women with acute hepatic porphyria (AHP) has historically been associated with significant morbidity. Clinical outcomes have been the focus of previous reports on porphyria and maternal health, with little data available on… read more here.
Keywords: acute hepatic; pregnancy; porphyria maternal; hepatic porphyria ... See more keywords
Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study
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DOI: 10.1111/liv.15090
Abstract: Upregulation of hepatic delta‐aminolevulinic acid synthase 1 with accumulation of potentially toxic heme precursors delta‐aminolevulinic acid and porphobilinogen is fundamental to the pathogenesis of acute hepatic porphyria. Aims: evaluate long‐term efficacy and safety of givosiran… read more here.
Keywords: acute hepatic; efficacy safety; givosiran acute; hepatic porphyria ... See more keywords