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Phenotypic screens as a renewed approach for drug discovery

Drug discovery today, 2013-11, Vol.18 (21-22), p.1067-1073

2013

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Ligand-Free Signaling of G-Protein-Coupled Receptors: Relevance to μ Opioid Receptors in Analgesia and Addiction

Molecules (Basel, Switzerland), 2022-09, Vol.27 (18), p.5826

2022

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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

The Lancet (British edition), 2017-10, Vol.390 (10104), p.1758-1768

2017

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Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease

Current topics in medicinal chemistry, 2014-02, Vol.14 (3), p.330-339

2014

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Identification of 53 compounds that block Ebola virus-like particle entry via a repurposing screen of approved drugs

Emerging microbes & infections, 2014-12, Vol.3 (12), p.e84-7

2014

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Chemical signatures and new drug targets for gametocytocidal drug development

Scientific reports, 2014-01, Vol.4 (1), p.3743-3743, Article 3743

2014

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Mutation Update for GNE Gene Variants Associated with GNE Myopathy

Human mutation, 2014-08, Vol.35 (8), p.915-926

2014

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Evaluation of chronic toxicity of cyclocreatine in beagle dogs after oral gavage administration for up to 23 weeks

Toxicology and applied pharmacology, 2021-11, Vol.430, p.115680-115680, Article 115680

2021

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Rapid identification of antifungal compounds against Exserohilum rostratum using high throughput drug repurposing screens

PloS one, 2013-08, Vol.8 (8), p.e70506

2013

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Use of an animal model of disease for toxicology enables identification of a juvenile no observed adverse effect level for cyclocreatine in creatine transporter deficiency

Regulatory toxicology and pharmacology, 2021-07, Vol.123, p.104939-104939, Article 104939

2021

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Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy

Journal of biomolecular screening, 2014-09, Vol.19 (8), p.1164-1173

2014

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A phenotypic compound screening assay for lysosomal storage diseases

Journal of biomolecular screening, 2014-01, Vol.19 (1), p.168-175

2014

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Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy

Molecular genetics and metabolism, 2017-09, Vol.122 (1-2), p.126-134

2017

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δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders

The Journal of biological chemistry, 2012-11, Vol.287 (47), p.39349-39360

2012

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Cyclocreatine treatment ameliorates the cognitive, autistic and epileptic phenotype in a mouse model of Creatine Transporter Deficiency

Scientific reports, 2020-10, Vol.10 (1), p.18361-18361, Article 18361

2020

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High throughput cell-based assay for identification of glycolate oxidase inhibitors as a potential treatment for Primary Hyperoxaluria Type 1

Scientific reports, 2016-09, Vol.6 (1), p.34060-34060, Article 34060

2016

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Evaluation of chronic toxicity of cyclocreatine, a creatine analog, in Sprague Dawley rat after oral gavage administration for up to 26 weeks

Regulatory toxicology and pharmacology, 2020-11, Vol.117, p.104750, Article 104750

2020

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Quantitative hydrophilic interaction chromatography–mass spectrometry analysis of N-acetylneuraminic acid and N-acetylmannosamine in human plasma

Journal of chromatography. B, Analytical technologies in the biomedical and life sciences, 2015-09, Vol.1000, p.105-111

2015

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A high-throughput screening assay for determining cellular levels of total tau protein

Current Alzheimer research, 2013-09, Vol.10 (7), p.679-687

2013

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A quantitative high throughput assay for identifying gametocytocidal compounds

Molecular and biochemical parasitology, 2013-03, Vol.188 (1), p.20-25

2013

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An open source pharma roadmap

PLoS medicine, 2017-04, Vol.14 (4), p.e1002276-e1002276

2017

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ERK and β-Arrestin Interaction: A Converging Point of Signaling Pathways for Multiple Types of Cell Surface Receptors

Journal of biomolecular screening, 2015-03, Vol.20 (3), p.341-349

2015

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Financing translation: analysis of the NCATS rare-diseases portfolio

Science translational medicine, 2015-02, Vol.7 (276), p.276ps3

2015

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High‐Throughput Phenotypic Screening of Human Astrocytes to Identify Compounds That Protect Against Oxidative Stress

Stem cells translational medicine, 2016-05, Vol.5 (5), p.613-627

2016

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Small molecule inhibition of group I p21-activated kinases in breast cancer induces apoptosis and potentiates the activity of microtubule stabilizing agents

Breast cancer research : BCR, 2015-04, Vol.17 (1), p.59, Article 59

2015

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Phenotypic screens as a renewed approach for drug discovery

Ligand-Free Signaling of G-Protein-Coupled Receptors: Relevance to μ Opioid Receptors in Analgesia and Addiction

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease

Identification of 53 compounds that block Ebola virus-like particle entry via a repurposing screen of approved drugs

Chemical signatures and new drug targets for gametocytocidal drug development

Mutation Update for GNE Gene Variants Associated with GNE Myopathy

Evaluation of chronic toxicity of cyclocreatine in beagle dogs after oral gavage administration for up to 23 weeks

Rapid identification of antifungal compounds against Exserohilum rostratum using high throughput drug repurposing screens

Use of an animal model of disease for toxicology enables identification of a juvenile no observed adverse effect level for cyclocreatine in creatine transporter deficiency

Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy

A phenotypic compound screening assay for lysosomal storage diseases

Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy

δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders

Cyclocreatine treatment ameliorates the cognitive, autistic and epileptic phenotype in a mouse model of Creatine Transporter Deficiency

High throughput cell-based assay for identification of glycolate oxidase inhibitors as a potential treatment for Primary Hyperoxaluria Type 1

Evaluation of chronic toxicity of cyclocreatine, a creatine analog, in Sprague Dawley rat after oral gavage administration for up to 26 weeks

Quantitative hydrophilic interaction chromatography–mass spectrometry analysis of N-acetylneuraminic acid and N-acetylmannosamine in human plasma

A high-throughput screening assay for determining cellular levels of total tau protein

A quantitative high throughput assay for identifying gametocytocidal compounds

An open source pharma roadmap

ERK and β-Arrestin Interaction: A Converging Point of Signaling Pathways for Multiple Types of Cell Surface Receptors

Financing translation: analysis of the NCATS rare-diseases portfolio

High‐Throughput Phenotypic Screening of Human Astrocytes to Identify Compounds That Protect Against Oxidative Stress

Small molecule inhibition of group I p21-activated kinases in breast cancer induces apoptosis and potentiates the activity of microtubule stabilizing agents

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