Zobrazeno 1 - 10
of 17
pro vyhledávání: '"Gabriel J. Robbie"'
Publikováno v:
CPT: Pharmacometrics & Systems Pharmacology, Vol 12, Iss 6, Pp 842-852 (2023)
Abstract Givosiran, an RNA interference‐based therapeutic, is a recent addition to the limited treatment armamentarium for acute hepatic porphyria (AHP). As a small interfering RNA that is selectively taken up in the liver, both the mechanism and t
Externí odkaz:
https://doaj.org/article/9993803bd27c4b6e8e4f30f0613223d0
Publikováno v:
Clinical Pharmacokinetics. 62:89-99
Publikováno v:
Bioanalysis.
Background: Measurement of plasma oxalate (POx) is challenging, but critical, for management of patients with primary hyperoxaluria type 1. A novel LC–MS/MS assay was developed, validated and used to quantify POx in patients with primary hyperoxalu
Autor:
Krishna Aluri, Scott Waldron, Karyn Schmidt, Maja M. Janas, Elena Castellanos-Rizaldos, Xiumin Liu, Michael Arciprete, Diane Ramsden, Jing Li, Vasant Jadhav, Robin McDougall, Christopher R. Brown, Bahru A. Habtemariam, Dale C. Guenther, Gabriel J. Robbie, Saket Agarwal, Muthiah Manoharan, Akshay Vaishnaw, Ivan Zlatev, Jeffrey Kurz, Jayaprakash K. Nair, Martin Maier, Saeho Chong, Sagar Agarwal, Christopher S. Theile, Steven Liou, Muthusamy Jayaraman, Varun Goel, Kevin Fitzgerald, Christopher MacLauchlin, Klaus Charisse, Joseph A Cichocki, Ju Liu, Yuanxin Xu, Peter F Smith, Jing-Tao Wu, Xuemei Zhang, Yongli Gu
Publikováno v:
Drug Metabolism and Disposition. 50:781-797
Conjugation of oligonucleotide therapeutics, including small interfering ribonucleic acids (siRNAs) or antisense oligonucleotides (ASOs) to N-acetylgalactosamine (GalNAc) ligands has become the primary strategy for hepatocyte-targeted delivery, and w
Autor:
Nader Najafian, Gabriel J. Robbie, Varun Goel, Anna Borodovsky, Prajakta S. Badri, Jae Kim, Bahru A. Habtemariam, Xuemin Jiang, Valerie A. Clausen
Publikováno v:
Clinical Pharmacokinetics. 60:365-378
Cemdisiran, an N-acetylgalactosamine (GalNAc) conjugated RNA interference (RNAi) therapeutic, is currently under development for the treatment of complement-mediated diseases by suppressing liver production of complement 5 (C5) protein. This study wa
Publikováno v:
JIMD Reports
Acute hepatic porphyria (AHP) is a family of rare, serious, and potentially life‐threatening metabolic disorders caused by mutations in genes encoding enzymes involved in hepatic heme biosynthesis. AHP is characterized by accumulation of neurotoxic
Autor:
Husain Attarwala, Valerie A. Clausen, Pushkal Garg, Varun Goel, Verena Karsten, Gabriel J. Robbie, Megan Melch, Akshay Vaishnaw, Bahru A. Habtemariam, John Vest, Marianne T. Sweetser
Publikováno v:
Clinical Pharmacology & Therapeutics. 109:372-382
Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid drug for the treatment of transthyretin (TTR)-mediated amyloidosis. This phase I, randomized, single-blind, placebo-controlled, single ascending dose st
Autor:
Varun Goel, Xiaoping Zhang, Gabriel J. Robbie, Jean-Francois Marier, Claudia Jomphe, Nathalie H. Gosselin
Publikováno v:
Nucleic Acid Therapeutics. 30:143-152
Hereditary transthyretin-mediated amyloidosis is an inherited, rapidly progressive, life-threatening disease caused by mutated transthyretin (TTR) protein. Patisiran is a small interfering RNA (siRNA) formulated in a lipid nanoparticle that inhibits
Autor:
Gabriel J. Robbie, Amy Simon, Valerie A. Clausen, Varun Goel, Jae B. Kim, Sagar Agarwal, Bahru A. Habtemariam
Publikováno v:
Clinical Pharmacology & Therapeutics. 108:63-72
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 subcu
Autor:
Shannon Gutierrez, Yuanxin Xu, Gabriel J. Robbie, Carrie Rocca, Samantha Chigas, James Butler, Saeho Chong, Yongli Gu, Sean Dennin, Joohwan Kim, Kirk Brown, Diana Najarian, Klaus Charisse
Publikováno v:
Bioanalysis. 11:1927-1939
Aim: The electrophoretic mobility shift assay (EMSA) was evaluated as an alternative to ultrafiltration (UF) to assess plasma protein binding (PPB) of small interfering RNAs (siRNA) and antisense oligonucleotides (ASO). Results & methodology: EMSA an