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Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

Clinical pharmacology and therapeutics, 2021-02, Vol.109 (2), p.372-382

2021

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Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis

Journal of clinical pharmacology, 2020-01, Vol.60 (1), p.37-49

2020

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A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

The New England journal of medicine, 2017-01, Vol.376 (1), p.41-51

2017

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Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria

Clinical pharmacology and therapeutics, 2020-07, Vol.108 (1), p.63-72

2020

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Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

The New England journal of medicine, 2013-08, Vol.369 (9), p.819-829

2013

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Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

Clinical pharmacokinetics, 2021-03, Vol.60 (3), p.365-378

2021

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A Drug‐Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver

Clinical pharmacology and therapeutics, 2021-11, Vol.110 (5), p.1250-1260

2021

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Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation

Clinical pharmacokinetics, 2023-10, Vol.62 (10), p.1509-1522

2023

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Correction to: Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

Clinical pharmacokinetics, 2022-06, Vol.61 (6), p.919-919

2022

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Nonclinical Pharmacokinetics and Absorption, Distribution, Metabolism, and Excretion of Givosiran, the First Approved N -Acetylgalactosamine-Conjugated RNA Interference Therapeutic

Drug metabolism and disposition, 2021-07, Vol.49 (7), p.572-580

2021

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Clinical and Preclinical Single-Dose Pharmacokinetics of VIR-2218, an RNAi Therapeutic Targeting HBV Infection

Drugs in R&D, 2021-12, Vol.21 (4), p.455-465

2021

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Quantification of oxalate by novel LC–MS/MS: assay development, validation and application in lumasiran clinical trials

Bioanalysis, 2023-05, Vol.15 (9), p.481-491

2023

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Structure–Activity Relationship (SAR) Optimization of 6‑(Indol-2-yl)pyridine-3-sulfonamides: Identification of Potent, Selective, and Orally Bioavailable Small Molecules Targeting Hepatitis C (HCV) NS4B

Journal of medicinal chemistry, 2014-03, Vol.57 (5), p.2121-2135

2014

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Discovery of novel HCV inhibitors: Synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B

Bioorganic & medicinal chemistry letters, 2013-07, Vol.23 (13), p.3947-3953

2013

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Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography-Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials: SA-PO571

Journal of the American Society of Nephrology, 2022-11, Vol.33 (11S), p.761-762

2022

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Cosilencing Intestinal Transglutaminase‑2 and Interleukin-15 Using Gelatin-Based Nanoparticles in an in Vitro Model of Celiac Disease

Molecular pharmaceutics, 2017-09, Vol.14 (9), p.3036-3044

2017

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In Vitro Drug-Drug Interaction Evaluation of GalNAc Conjugated siRNAs Against CYP450 Enzymes and Transporters

Drug metabolism and disposition, 2019-10, Vol.47 (10), p.1183-1194

2019

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Biochemical characterization of the first essential two-component signal transduction system from Staphylococcus aureus and Streptococcus pneumoniae

Journal of molecular microbiology and biotechnology, 2003-01, Vol.5 (4), p.252-260

2003

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Oligonucleotide quantification and metabolite profiling by high-resolution and accurate mass spectrometry

Bioanalysis, 2019-11, Vol.11 (21), p.1967-1980

2019

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Stereochemical Analysis of the Reaction Catalyzed by Human Protein Geranylgeranyl Transferase

Biochemistry (Easton), 2001-04, Vol.40 (13), p.3920-3930

2001

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2021 White Paper on Recent Issues in Bioanalysis: ISR for Biomarkers, Liquid Biopsies, Spectral Cytometry, Inhalation/Oral & Multispecific Biotherapeutics, Accuracy/LLOQ for Flow Cytometry

Bioanalysis, 2022-05, Vol.14 (10), p.627-692

2022

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Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

A Drug‐Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver

First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement

Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation

Correction to: Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

Nonclinical Pharmacokinetics and Absorption, Distribution, Metabolism, and Excretion of Givosiran, the First Approved N -Acetylgalactosamine-Conjugated RNA Interference Therapeutic

Clinical and Preclinical Single-Dose Pharmacokinetics of VIR-2218, an RNAi Therapeutic Targeting HBV Infection

Quantification of oxalate by novel LC–MS/MS: assay development, validation and application in lumasiran clinical trials

Structure–Activity Relationship (SAR) Optimization of 6‑(Indol-2-yl)pyridine-3-sulfonamides: Identification of Potent, Selective, and Orally Bioavailable Small Molecules Targeting Hepatitis C (HCV) NS4B

Discovery of novel HCV inhibitors: Synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B

Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography-Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials: SA-PO571

Cosilencing Intestinal Transglutaminase‑2 and Interleukin-15 Using Gelatin-Based Nanoparticles in an in Vitro Model of Celiac Disease

In Vitro Drug-Drug Interaction Evaluation of GalNAc Conjugated siRNAs Against CYP450 Enzymes and Transporters

Biochemical characterization of the first essential two-component signal transduction system from Staphylococcus aureus and Streptococcus pneumoniae

Oligonucleotide quantification and metabolite profiling by high-resolution and accurate mass spectrometry

Stereochemical Analysis of the Reaction Catalyzed by Human Protein Geranylgeranyl Transferase

2019 White Paper on Recent Issues in Bioanalysis: Chromatographic Assays (Part 1 – Innovation in Small Molecules and Oligonucleotides & Mass Spectrometric Method Development Strategies for Large Molecule Bioanalysis)

2021 White Paper on Recent Issues in Bioanalysis: ISR for Biomarkers, Liquid Biopsies, Spectral Cytometry, Inhalation/Oral & Multispecific Biotherapeutics, Accuracy/LLOQ for Flow Cytometry

Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles

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