Results 111 to 120 of about 1,115 (145)

Advancing hypertension management: the role of zilebesiran as an siRNA therapeutic agent. [PDF]

Ann Med Surg (Lond)
Siddiqui E, Siddiqui AH, Moeed A, Laique F, Najeeb H, Al Hasibuzzaman M.   +5 more
europepmc   +1 more source

Some of the next articles are maybe not open access.

Related searches:

Givosiran in acute intermittent porphyria: A personalized medicine approach

Molecular Genetics and Metabolism, 2022
In patients with acute intermittent porphyria (AIP), induction of delta aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor accumulation that may cause recurring acute attacks. In a recent phase III trial, givosiran significantly reduced the attack rate in severe AIP patients.
Caroline Schmitt, Katell Peoc'H, Hervé Puy   +2 more
exaly   +3 more sources

Givosiran for the treatment of acute hepatic porphyria

Expert Review of Clinical Pharmacology, 2022
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 porphobilinogen, caused by enhanced activity of hepatic aminolevulinate synthase 1 (ALAS1), is associated ...
Paolo Ventura, Paolo Ventura, Andrea Ricci   +2 more
exaly   +3 more sources

Givosiran: First Approval

Drugs, 2020
Givosiran (Givlaari™) is an aminolevulinate synthase 1 (ALAS1)-directed small interfering RNA (siRNA) covalently linked to a ligand to enable specific delivery of the siRNA to hepatocytes. This results in downregulation of ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels that are associated with acute ...
Scott Lesley J
exaly   +3 more sources

Givosiran for Acute Intermittent Porphyria

New England Journal of Medicine, 2020
Alberto Ortiz Arduan, Elena Gomá-Garcés   +1 more
exaly   +3 more sources

Givosiran to treat acute porphyria

Drugs of Today, 2021
Porphyrias are a family of rare diseases chiefly due to inborn errors of heme biosynthesis. The porphyrias are generally characterized either by the main site of overproduction of heme precursors (hepatic or erythropoietic) or the main clinical manifestations (acute or cutaneous). The regulation of 5- (or δ)-aminolevulinic acid synthase 1 (ALAS1) plays
A, Honor, S R, Rudnick, H L, Bonkovsky
openaire   +2 more sources

Givosiran: A Review in Acute Hepatic Porphyria

Drugs, 2021
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 phase 3 ENVISION trial, givosiran significantly reduced the annualized rate of composite porphyria attacks (i.e.
openaire   +2 more sources

Population Pharmacokinetic Analysis of the RNAi Therapeutic Givosiran in Patients with Acute Hepatic Porphyria

Clinical Pharmacokinetics, 2023
Givosiran, approved for the treatment of acute hepatic porphyria (AHP), is the first subcutaneously administered RNAi therapeutic. This analysis was undertaken to describe the plasma pharmacokinetics (PK) of givosiran and its active metabolite, AS(N-1)3' givosiran, and to identify factors that contribute to intersubject PK variability.A population PK ...
Megan, Melch, Jongtae, Lee, Claudia, Jomphe, Gabriel J, Robbie   +3 more
openaire   +2 more sources

Givosiran, a novel treatment for acute hepatic porphyrias

Expert Review of Precision Medicine and Drug Development, 2020
Acute hepatic porphyrias (AHPs) are a group of rare genetic disorders that affect the enzymes of the heme biosynthetic pathway.
Manish Thapar, Sean Rudnick, Herbert L. Bonkovsky   +2 more
openaire   +1 more source