D. Norris

发表

Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys.

B. Monia, Shin-Young Park, S. Henry, 2013, Nucleic acid therapeutics.

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

R. Finkel, F. Rigo, D. Vivo, 2016, The Lancet.

Characterization of target mRNA reduction through in situ RNA hybridization in multiple organ systems following systemic antisense treatment in animals.

Shuling Guo, B. Monia, D. Norris, 2013, Nucleic acid therapeutics.

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides.

C. Bennett, R. Yu, R. Geary, 2015, Advanced drug delivery reviews.

Targeting Huntingtin Expression in Patients with Huntington's Disease.

R. Barker, S. Tabrizi, B. Leavitt, 2019, The New England journal of medicine.

2018 White Paper on Recent Issues in Bioanalysis: 'A global bioanalytical community perspective on last decade of incurred samples reanalysis (ISR)' (Part 1 - small molecule regulated bioanalysis, small molecule biomarkers, peptides & oligonucleotide bioanalysis).

Sam H Haidar, Gustavo Mendes Lima Santos, Nicola C Hughes, 2018, Bioanalysis.

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

A. Pestronk, D. Schoenfeld, P. Andres, 2013, The Lancet Neurology.

Assessment of the Drug Interaction Potential of Unconjugated and GalNAc3-Conjugated 2′-MOE-ASOs

E. Hurh, S. Henry, R. Yu, 2017, Molecular therapy. Nucleic acids.

Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides

M. Benson, Shuling Guo, B. Monia, 2016, Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis.

Bioanalysis considerations on the pharmacokinetic evaluation of antisense therapeutics.

R. Yu, D. Norris, Sarah Greenlee, 2019, Bioanalysis.

5,5'- and 6,6'-dialkyl-5,6-dihydro-1H-pyridin-2-ones as potent inhibitors of HCV NS5B polymerase.

Matthew A. Lardy, C. Kissinger, Qiang Zhao, 2009, Bioorganic & medicinal chemistry letters.

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

F. Rigo, J. Matson, P. Jafar-Nejad, 2020, bioRxiv.

Edinburgh Research Explorer Targeting Huntingtin Expression in Patients with Huntington’s Disease

R. Barker, S. Tabrizi, B. Leavitt, 2019 .

A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration

I. Nestorov, M. Monine, D. Norris, 2021, Journal of Pharmacokinetics and Pharmacodynamics.

Determining the absolute surface hydrophobicity of microparticulates using thin layer wicking.

D. Norris, P. Sinko, M. Labib, 1999, Journal of controlled release : official journal of the Controlled Release Society.

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.

T. Crawford, S. Paushkin, F. Rigo, 2019, The Journal of clinical investigation.

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Y. Hua, A. Krainer, N. York, 2014, The Journal of Pharmacology and Experimental Therapeutics.

Results of an Open-Label, Escalating Dose Study To Assess the Safety, Tolerability, and Dose Range Finding of a Single Intrathecal Dose of ISIS-SMNRx in Patients with Spinal Muscular Atrophy (S36.002)

S. Iannaccone, K. Swoboda, D. Vivo, 2013 .

Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations

S. Henry, R. Geary, D. Norris, 2017, Journal of clinical pharmacology.

Discovery of tricyclic 5,6-dihydro-1H-pyridin-2-ones as novel, potent, and orally bioavailable inhibitors of HCV NS5B polymerase.

P. A. Thompson, J. Khandurina, C. Kissinger, 2009, Bioorganic & medicinal chemistry letters.

Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002)

S. Tabrizi, B. Leavitt, S. Schobel, 2018 .

Discovery and Early Clinical Development of ISIS-HTTRx, the First HTT-Lowering Drug to Be Tested in Patients with Huntington’s Disease (PL01.002)

S. Tabrizi, B. Leavitt, B. Landwehrmeyer, 2016 .

Results of the first‐in‐human, randomized, double‐blind, placebo‐controlled phase 1b study of lumbar intrathecal bolus administrations of antisense oligonucleotide (ISIS 814907; BIIB080) targeting tau mRNA in patients with mild Alzheimer’s disease

C. Mummery, L. Mignon, D. Norris, 2021, Alzheimer's & Dementia.

Pyrrolo[1,2-b]pyridazin-2-ones as potent inhibitors of HCV NS5B polymerase.

Jingjing Zhao, Matthew A. Lardy, Frank Ruebsam, 2008, Bioorganic & medicinal chemistry letters.

5,6-Dihydro-1H-pyridin-2-ones as potent inhibitors of HCV NS5B polymerase.

Yuefen Zhou, S. Webber, D. Murphy, 2009, Bioorganic & medicinal chemistry letters.

Characterization of the Regional Intestinal Kinetics of Drug Efflux in Rat and Human Intestine and in Caco-2 Cells

Patrick J. Sinko, Ailan Guo, A. Guo, 1998, Pharmaceutical Research.

Development of predictive pharmacokinetic simulation models for drug discovery.

P J Sinko, G. Grass, D. Norris, 2000, Journal of controlled release : official journal of the Controlled Release Society.

A Selective Inhibitor of Human C-reactive Protein Translation Is Efficacious In Vitro and in C-reactive Protein Transgenic Mice and Humans

M. Graham, R. Crooke, A. Szalai, 2012, Molecular therapy. Nucleic acids.

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

Jacqueline Montes, S. Iannaccone, K. Swoboda, 2016, Neurology.

A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy

Mirjam N Trame, L. Lesko, I. Nestorov, 2018, CPT: pharmacometrics & systems pharmacology.

Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

S. Subramony, T. Ashizawa, J. Statland, 2023, The Lancet Neurology.

Predictive Dose-Based Estimation of Systemic Exposure Multiples in Mouse and Monkey Relative to Human for Antisense Oligonucleotides With 2′-O-(2-Methoxyethyl) Modifications

S. Henry, R. Yu, R. Geary, 2015, Molecular therapy. Nucleic acids.

Design of the First-in-Human Study of IONIS-MAPTRx, a Tau-lowering Antisense Oligonucleotide, in Patients With Alzheimer Disease (S2.006)

L. Mignon, B. Fitzsimmons, D. Norris, 2018 .

EVALUATION OF BATCH-TO-BATCH AND MANUFACTURER-TO- MANUFACTURER VARIABILITY IN THE PHYSICAL PROPERTIES OF TALC AND STEARIC ACID

D. Norris, Melissa Keeney, D. Phadke, 1994 .

Antisense oligonucleotide inhibition of cholesteryl ester transfer protein enhances RCT in hyperlipidemic, CETP transgenic, LDLr−/− mice

Richard G. Lee, M. Graham, R. Crooke, 2013, Journal of Lipid Research.

Antisense Oligonucleotide Inhibition of Cholesteryl Ester Transfer Protein Enhances Reverse Cholesterol Transport in Hyperlipidemic CETP Transgenic , LDLr-/-Mice

Richard G. Lee, M. Graham, R. Crooke, 2013 .

Hexahydro-pyrrolo- and hexahydro-1H-pyrido[1,2-b]pyridazin-2-ones as potent inhibitors of HCV NS5B polymerase.

C. Kissinger, Qiang Zhao, Yuefen Zhou, 2008, Bioorganic & medicinal chemistry letters.

4-(1,1-Dioxo-1,4-dihydro-1lambda6-benzo[1,4]thiazin-3-yl)-5-hydroxy-2H-pyridazin-3-ones as potent inhibitors of HCV NS5B polymerase.

Matthew A. Lardy, C. Kissinger, Qing Han, 2008, Bioorganic & medicinal chemistry letters.

Results of a Phase 2 Open-Label Study of ISIS-SMNRx in Patients with Infantile (Type 1) Spinal Muscular Atrophy (S6.003)

R. Finkel, D. Vivo, J. Montes, 2014 .

Structure-based design, synthesis, and biological evaluation of 1,1-dioxoisothiazole and benzo[b]thiophene-1,1-dioxide derivatives as novel inhibitors of hepatitis C virus NS5B polymerase.

Qiang Zhao, Helen McGuire, Alberto Gobbi, 2008, Bioorganic & medicinal chemistry letters.

Translating Antisense Technology into a Treatment for Huntington's Disease.

M. Gleichmann, C. Bennett, D. Norris, 2018, Methods in molecular biology.

Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial

C. Mummery, J. Rinne, M. Jonsson, 2023, Nature Medicine.

Novel HCV NS5B polymerase inhibitors derived from 4-(1',1'-dioxo-1',4'-dihydro-1'lambda(6)-benzo[1',2',4']thiadiazin-3'-yl)-5-hydroxy-2H-pyridazin-3-ones. Part 3: Further optimization of the 2-, 6-, and 7'-substituents and initial pharmacokinetic assessments.

C. Kissinger, Qiang Zhao, Yuefen Zhou, 2008, Bioorganic & medicinal chemistry letters.

Novel HCV NS5B polymerase inhibitors derived from 4-(1',1'-dioxo-1',4'-dihydro-1'lambda6-benzo[1',2',4']thiadiazin-3'-yl)-5-hydroxy-2H-pyridazin-3-ones: Part 4. Optimization of DMPK properties.

Matthew A. Lardy, Yuefen Zhou, S. Webber, 2008, Bioorganic & medicinal chemistry letters.

Effect of size, surface charge, and hydrophobicity on the translocation of polystyrene microspheres through gastrointestinal mucin

Patrick J. Sinko, D. Norris, P. Sinko, 1997 .

Interim Results of a Phase 2 Clinical Study of Nusinersen (ISIS-SMNRx) in Patients with Infantile-Onset Spinal Muscular Atrophy (P5.004)

R. Finkel, F. Rigo, D. Vivo, 2016 .