Givosiran (Givlaari®) is an δ-aminolevulinic acid synthase 1 (ALAS1)-directed small
interfering RNA (siRNA) approved for the treatment of acute hepatic porphyria (AHP). In the …

Recent years have seen unprecedented activity in the development of RNA-silencing
oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and …

Abstract Background & Aims Upregulation of hepatic delta‐aminolevulinic acid synthase 1
with accumulation of potentially toxic heme precursors delta‐aminolevulinic acid and …

Background & Aims Acute hepatic porphyria (AHP) is caused by defects in hepatic heme
biosynthesis, leading to disabling acute neurovisceral attacks and chronic symptoms. In …

Acute intermittent porphyria (AIP) is a rare metabolic disease caused by mutations within the
hydroxymethylbilane synthase gene. Previous studies have reported increased levels of …

Background In patients with acute intermittent porphyria (AIP), induction of delta
aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor accumulation that may …

Givosiran is a novel approach to treat patients with acute intermittent porphyrias (AIP) by
silencing of∂-ALA-synthase 1, the first enzyme of heme biosynthesis in the liver. We …

Introduction Homocysteine is a sulfur-containing amino acid formed in the intermediary
metabolism of methionine. Amino acid metabolism and heme biosynthesis pathways are …

Acute intermittent porphyria (AIP) is a rare genetic metabolic disease caused by a specific
enzyme dysfunction in the hepatic heme biosynthesis pathway. Besides the obvious clinical …

Given its remarkable property to easily switch between different oxidative states, iron is
essential in countless cellular functions which involve redox reactions. At the same time …