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The clinical progress of mRNA vaccines and immunotherapies

Nature biotechnology, 2022-06, Vol.40 (6), p.840-854

2022

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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

The New England journal of medicine, 2019-09, Vol.381 (10), p.933-944

2019

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A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

Genetics in medicine, 2016-01, Vol.18 (1), p.73-81

2016

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A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA

Molecular genetics and metabolism, 2016-07, Vol.118 (3), p.198-205

2016

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Inhibition of fatty acid amide hydrolase produces analgesia by multiple mechanisms

British journal of pharmacology, 2006-05, Vol.148 (1), p.102-113

2006

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Phase 1 Single‐ and Multiple‐Ascending‐Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG‐348, a First‐in‐Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers

Clinical pharmacology in drug development, 2019-02, Vol.8 (2), p.246-259

2019

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Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions

Molecular genetics and metabolism, 2016-06, Vol.118 (2), p.65-69

2016

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Radiosynthesis and biodistribution of a histamine H3 receptor antagonist 4-[3-(4-piperidin-1-yl-but-1-ynyl)-[11C]benzyl]-morpholine: evaluation of a potential PET ligand

Nuclear medicine and biology, 2006-08, Vol.33 (6), p.801-810

2006

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A novel LC-MS/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients

Bioanalysis, 2016-02, Vol.8 (4), p.285-295

2016

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Behavioral Characterization of Mice Lacking Histamine H3 Receptors

Molecular pharmacology, 2002-08, Vol.62 (2), p.389

2002

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Characterization of Soluble Forms of Nonchimeric Type V Adenylyl Cyclases

Proceedings of the National Academy of Sciences - PNAS, 1997-04, Vol.94 (7), p.2915-2920

1997

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Patient Voice in Rare Disease Drug Development and Endpoints

Therapeutic innovation & regulatory science, 2017-03, Vol.51 (2), p.257-263

2017

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In-vitro and in-vivo characterization of JNJ-7925476, a novel triple monoamine uptake inhibitor

European journal of pharmacology, 2008-06, Vol.587 (1), p.141-146

2008

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Pyrrolidino-tetrahydroisoquinolines as potent dual H3 antagonist and serotonin transporter inhibitors

Bioorganic & medicinal chemistry letters, 2007-05, Vol.17 (9), p.2603-2607

2007

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A high-throughput-compatible assay for determining the activity of fatty acid amide hydrolase

Analytical biochemistry, 2003-07, Vol.318 (2), p.270-275

2003

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Transmodulation of Epidermal Growth Factor Receptor Function by Cyclic AMP-dependent Protein Kinase

The Journal of biological chemistry, 1999-05, Vol.274 (20), p.14067-14073

1999

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Population Pharmacokinetics and Pharmacodynamics of AG-519, a Pyruvate Kinase Activator for the Treatment of Pyruvate Kinase Deficiency, in Human Healthy Volunteers

Blood, 2016-12, Vol.128 (22), p.1263-1263

2016

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Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of AG-519, an Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects

Blood, 2016-12, Vol.128 (22), p.1264-1264

2016

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Characterization of Metabolic Response to AG-348, an Allosteric Activator of Red Cell Pyruvate Kinase, in Healthy Volunteers and Pyruvate Kinase Deficiency Patients

Blood, 2016-12, Vol.128 (22), p.2452-2452

2016

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A simple stopped assay for fatty acid amide hydrolase avoiding the use of a chloroform extraction phase

Journal of biochemical and biophysical methods, 2004-08, Vol.60 (2), p.171-177

2004

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Immunogenicity of idursulfase and clinical outcomes in very young patients

Orphanet journal of rare diseases, 2015-04, Vol.10

2015

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Identification of an Intramolecular Interaction between Small Regions in Type V Adenylyl Cyclase that Influences Stimulation of Enzyme Activity by Gsα

Proceedings of the National Academy of Sciences - PNAS, 1997-09, Vol.94 (18), p.9602-9607

1997

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Behavioral Characterization of Mice Lacking Histamine H 3 Receptors

Molecular pharmacology, 2002-08, Vol.62 (2), p.389-397

2002

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Antibody response to investigational intrathecal enzyme replacement therapy with idursulfase-IT in pediatric Hunter syndrome patients with cognitive impairment

Molecular genetics and metabolism, 2015-02, Vol.114 (2), p.S53-S53

2015

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The clinical progress of mRNA vaccines and immunotherapies

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA

Inhibition of fatty acid amide hydrolase produces analgesia by multiple mechanisms

Phase 1 Single‐ and Multiple‐Ascending‐Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG‐348, a First‐in‐Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers

Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions

Radiosynthesis and biodistribution of a histamine H3 receptor antagonist 4-[3-(4-piperidin-1-yl-but-1-ynyl)-[11C]benzyl]-morpholine: evaluation of a potential PET ligand

A novel LC-MS/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients

Behavioral Characterization of Mice Lacking Histamine H3 Receptors

Characterization of Soluble Forms of Nonchimeric Type V Adenylyl Cyclases

Patient Voice in Rare Disease Drug Development and Endpoints

In-vitro and in-vivo characterization of JNJ-7925476, a novel triple monoamine uptake inhibitor

Pyrrolidino-tetrahydroisoquinolines as potent dual H3 antagonist and serotonin transporter inhibitors

A high-throughput-compatible assay for determining the activity of fatty acid amide hydrolase

Transmodulation of Epidermal Growth Factor Receptor Function by Cyclic AMP-dependent Protein Kinase

Population Pharmacokinetics and Pharmacodynamics of AG-519, a Pyruvate Kinase Activator for the Treatment of Pyruvate Kinase Deficiency, in Human Healthy Volunteers

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of AG-519, an Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects

Characterization of Metabolic Response to AG-348, an Allosteric Activator of Red Cell Pyruvate Kinase, in Healthy Volunteers and Pyruvate Kinase Deficiency Patients

A simple stopped assay for fatty acid amide hydrolase avoiding the use of a chloroform extraction phase

Immunogenicity of idursulfase and clinical outcomes in very young patients

Identification of an Intramolecular Interaction between Small Regions in Type V Adenylyl Cyclase that Influences Stimulation of Enzyme Activity by Gsα

Behavioral Characterization of Mice Lacking Histamine H 3 Receptors

Antibody response to investigational intrathecal enzyme replacement therapy with idursulfase-IT in pediatric Hunter syndrome patients with cognitive impairment

Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study

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