Results 121 to 130 of about 1,115 (145)
Some of the next articles are maybe not open access.
Journal of Inherited Metabolic Disease, 2021
AbstractAcute intermittent porphyria (AIP) is a rare metabolic disease caused by mutations within the hydroxymethylbilane synthase gene. Previous studies have reported increased levels of plasma total homocysteine (tHcy) in symptomatic AIP patients. In this study, we present long‐term data for tHcy and related parameters for an AIP patient cohort (n ...
Jordi To‐Figueras +9 more
openaire +2 more sources
AbstractAcute intermittent porphyria (AIP) is a rare metabolic disease caused by mutations within the hydroxymethylbilane synthase gene. Previous studies have reported increased levels of plasma total homocysteine (tHcy) in symptomatic AIP patients. In this study, we present long‐term data for tHcy and related parameters for an AIP patient cohort (n ...
Jordi To‐Figueras +9 more
openaire +2 more sources
Givosiran — Running RNA Interference to Fight Porphyria Attacks
New England Journal of Medicine, 2020The porphyrias are a group of rare and ultra-rare devastating disorders of heme biosynthesis. The majority of these disorders are inherited, and patients present with disabling symptoms that have a...
openaire +2 more sources
Clinical Pharmacology & Therapeutics, 2020
Givosiran is a small interfering ribonucleic acid agent that was recently approved in the United States for the treatment of acute hepatic porphyria (AHP). This phase I study evaluated the safety, pharmacokinetic, and pharmacodynamic profile of subcutaneously (SC) administered givosiran in patients with acute intermittent porphyria, the most common AHP
Sagar, Agarwal +6 more
openaire +2 more sources
Givosiran is a small interfering ribonucleic acid agent that was recently approved in the United States for the treatment of acute hepatic porphyria (AHP). This phase I study evaluated the safety, pharmacokinetic, and pharmacodynamic profile of subcutaneously (SC) administered givosiran in patients with acute intermittent porphyria, the most common AHP
Sagar, Agarwal +6 more
openaire +2 more sources
Givosiran als neue therapeutische Option
Gastro-News, 2021Heiner Wedemeyer +2 more
openaire +1 more source
Acute intermittent porphyria: acute flare-up resolved with givosiran
Medicina Clínica (English Edition), 2023Andrea María, Balado González +2 more
openaire +2 more sources

