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Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration

Pediatric neurology, 2017-02, Vol.67, p.23-35

2017

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A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

The Journal of clinical investigation, 2023-01, Vol.133 (2), p.1-13

2023

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CREB: A Stimulus-Induced Transcription Factor Activated by A Diverse Array of Extracellular Signals

Annual review of biochemistry, 1999-01, Vol.68 (1), p.821-861

1999

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Effects of AMG 145 on Low-Density Lipoprotein Cholesterol Levels: Results From 2 Randomized, Double-Blind, Placebo-Controlled, Ascending-Dose Phase 1 Studies in Healthy Volunteers and Hypercholesterolemic Subjects on Statins

Journal of the American College of Cardiology, 2012-11, Vol.60 (19), p.1888-1898

2012

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Best practices for the use of intracerebroventricular drug delivery devices

Molecular genetics and metabolism, 2018-07, Vol.124 (3), p.184-188

2018

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Hypocalcaemia in patients with metastatic bone disease treated with denosumab

European journal of cancer (1990), 2015-09, Vol.51 (13), p.1812-1821

2015

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Longitudinal natural history of pediatric subjects affected with mucopolysaccharidosis IIIB

The Journal of pediatrics, 2022-10, Vol.249, p.50-58.e2

2022

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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Molecular therapy, 2022-07, Vol.30 (7), p.2416-2428

2022

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Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

Pediatric research, 2015-12, Vol.78 (6), p.717-722

2015

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Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial

Clinical therapeutics, 2015-05, Vol.37 (5), p.1012-1021.e6

2015

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Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Orphanet journal of rare diseases, 2017-06, Vol.12 (1), p.117-117, Article 117

2017

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Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Molecular genetics and metabolism, 2016-09, Vol.119 (1-2), p.131-143

2016

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Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome

Clinical pharmacokinetics, 2014-12, Vol.53 (12), p.1137-1147

2014

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CREB Transcriptional Activity in Neurons Is Regulated by Multiple, Calcium-Specific Phosphorylation Events

Neuron (Cambridge, Mass.), 2002-04, Vol.34 (2), p.221-233

2002

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Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study

American journal of medical genetics. Part A, 2015-10, Vol.167A (10), p.2272-2281

2015

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Calcium Regulation of Neuronal Gene Expression

Proceedings of the National Academy of Sciences - PNAS, 2001-09, Vol.98 (20), p.11024-11031

2001

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Ca2+ Influx Regulates BDNF Transcription by a CREB Family Transcription Factor-Dependent Mechanism

Neuron (Cambridge, Mass.), 1998-04, Vol.20 (4), p.709-726

1998

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Effects of AMG 145 on Low-Density Lipoprotein Cholesterol Levels

Journal of the American College of Cardiology, 2012-11, Vol.60 (19), p.1888-1898

2012

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Tolerability of High-Volume Subcutaneous Injections of a Viscous Placebo Buffer: A Randomized, Crossover Study in Healthy Subjects

AAPS PharmSciTech, 2015-10, Vol.16 (5), p.1101-1107

2015

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AMPK-Dependent Degradation of TXNIP upon Energy Stress Leads to Enhanced Glucose Uptake via GLUT1

Molecular cell, 2013-03, Vol.49 (6), p.1167-1175

2013

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Effects of Evolocumab on Lipoprotein Particles & Subclasses in Hypercholesterolemic & Heterozygous Familial Hypercholesterolemia Subjects on Statin

Journal of clinical lipidology, 2014-05, Vol.8 (3), p.350-351

2014

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Design and rationale of the study programs for BMN 250, a novel enzyme replacement therapy (ERT) for Sanfilippo syndrome type B

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S105-S106

2016

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AMG 145, a Fully Human Monoclonal Antibody Against PCSK9, Reduces LDL-C in Healthy Volunteers and Patients on Stable Doses of Statins

Journal of clinical lipidology, 2012-05, Vol.6 (3), p.286-287

2012

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Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration

A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

CREB: A Stimulus-Induced Transcription Factor Activated by A Diverse Array of Extracellular Signals

Effects of AMG 145 on Low-Density Lipoprotein Cholesterol Levels: Results From 2 Randomized, Double-Blind, Placebo-Controlled, Ascending-Dose Phase 1 Studies in Healthy Volunteers and Hypercholesterolemic Subjects on Statins

Best practices for the use of intracerebroventricular drug delivery devices

Hypocalcaemia in patients with metastatic bone disease treated with denosumab

Longitudinal natural history of pediatric subjects affected with mucopolysaccharidosis IIIB

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome

CREB Transcriptional Activity in Neurons Is Regulated by Multiple, Calcium-Specific Phosphorylation Events

Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study

Calcium Regulation of Neuronal Gene Expression

Ca2+ Influx Regulates BDNF Transcription by a CREB Family Transcription Factor-Dependent Mechanism

Effects of AMG 145 on Low-Density Lipoprotein Cholesterol Levels

Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B

Tolerability of High-Volume Subcutaneous Injections of a Viscous Placebo Buffer: A Randomized, Crossover Study in Healthy Subjects

AMPK-Dependent Degradation of TXNIP upon Energy Stress Leads to Enhanced Glucose Uptake via GLUT1

Effects of Evolocumab on Lipoprotein Particles & Subclasses in Hypercholesterolemic & Heterozygous Familial Hypercholesterolemia Subjects on Statin

Design and rationale of the study programs for BMN 250, a novel enzyme replacement therapy (ERT) for Sanfilippo syndrome type B

AMG 145, a Fully Human Monoclonal Antibody Against PCSK9, Reduces LDL-C in Healthy Volunteers and Patients on Stable Doses of Statins

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