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World Federation of Hemophilia Gene Therapy Registry

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-07, Vol.26 (4), p.563-564

2020

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Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)-10-year follow-up of patients who previously participated in an MPS VI survey study

American journal of medical genetics. Part A, 2014-08, Vol.164A (8), p.1953-1964

2014

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A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences

Molecular genetics and metabolism reports, 2019-12, Vol.21, p.100507-100507, Article 100507

2019

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Effects of Food Intake on the Relative Bioavailability of Amifampridine Phosphate Salt in Healthy Adults

Clinical therapeutics, 2015-07, Vol.37 (7), p.1555-1563

2015

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Eosinophilic myocarditis in patients awaiting heart transplantation

Critical care medicine, 2004-03, Vol.32 (3), p.714-721

2004

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Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)

Journal of inborn errors of metabolism and screening, 2018-01, Vol.6

2018

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UANTIFYING PREFERENCES FOR UREA CYCLE DISORDER TREATMENTS USING A DISCRETE CHOICE EXPERIMENT

Molecular genetics and metabolism, 2023-03, Vol.138 (3), p.107470, Article 107470

2023

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Pulmonary function tests (maximum inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator use in late-onset Pompe disease

Neuromuscular disorders : NMD, 2016-02, Vol.26 (2), p.136-145

2016

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Quantifying preferences for urea cycle disorder treatments using a discrete choice experiment

Journal of medical economics, 2024, Vol.27 (1), p.506-517

2024

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Immediate-release/extended-release amantadine (OS320) to treat Parkinson's disease with levodopa-induced dyskinesia: Analysis of the randomized, controlled ALLAY-LID studies

Parkinsonism & related disorders, 2022-03, Vol.96, p.65-73

2022

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Growth charts for individuals with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S88-S89

2014

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Guidelines for sample collection and handling from the biomarker working group for the mucopolysaccharidoses

Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S43-S43

2014

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Long-term galsulfase treatment associated with improved survival of patients with mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): 15 year follow-up from the survey study

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S56-S56

2017

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Sub-analysis of long-term elosulfase alfa treatment outcomes in adults with Morquio syndrome type A

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S67-S68

2017

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Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study

Journal of inborn errors of metabolism and screening, 2018-01, Vol.6, p.232640981875580

2018

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The Morquio A Registry Study (MARS): Improving the understanding of Morquio syndrome type A

Molecular genetics and metabolism, 2015-02, Vol.114 (2), p.S49-S49

2015

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Keratan sulfate (KS) analysis in patients with Morquio syndrome type A: LC-MS/MS analysis of KS disaccharides demonstrates that urine is a better source than plasma to monitor dynamic change in KS

Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S77-S77

2014

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Galsulfase for mucopolysaccharidosis type VI: analysis of clinical data since 2000

Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S52-S53

2014

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Orphan drugs. BioMarin Europe replies

BMJ (Online), 2010-12, Vol.341, p.c7006

2010

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Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux–Lamy Syndrome)

JIMD Reports, Volume 18, 2015, Vol.18, p.1-11

2015

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BioMarin Europe replies

BMJ, 2010-12, Vol.341 (dec07 2), p.c7006-c7006

2010

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Cardiovascular Safety of the Oral Controlled Absorption System (OCAS) Formulation of Tamsulosin Compared to the Modified Release (MR) Formulation

European urology supplements : official journal of the European Association of Urology, 2005-02, Vol.4 (2), p.53-60

2005

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Comparison of Vascular α1-Adrenoceptor Antagonism of Tamsulosin in Oral Controlled Absorption System (OCAS) and Modified Release (MR) Formulations

European urology supplements : official journal of the European Association of Urology, 2005-02, Vol.4 (2), p.45-52

2005

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Inhibition of Graft Coronary Arteriosclerosis after Heart Transplantation

The American surgeon, 2004-10, Vol.70 (10), p.833-840

2004

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Comparison of Vascular α 1-Adrenoceptor Antagonism of Tamsulosin in Oral Controlled Absorption System (OCAS) and Modified Release (MR) Formulations

European urology supplements : official journal of the European Association of Urology, 2005, Vol.4 (2), p.45-52

2005

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World Federation of Hemophilia Gene Therapy Registry

Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)-10-year follow-up of patients who previously participated in an MPS VI survey study

A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences

Effects of Food Intake on the Relative Bioavailability of Amifampridine Phosphate Salt in Healthy Adults

Eosinophilic myocarditis in patients awaiting heart transplantation

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)

UANTIFYING PREFERENCES FOR UREA CYCLE DISORDER TREATMENTS USING A DISCRETE CHOICE EXPERIMENT

Pulmonary function tests (maximum inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator use in late-onset Pompe disease

Quantifying preferences for urea cycle disorder treatments using a discrete choice experiment

Immediate-release/extended-release amantadine (OS320) to treat Parkinson's disease with levodopa-induced dyskinesia: Analysis of the randomized, controlled ALLAY-LID studies

Growth charts for individuals with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Guidelines for sample collection and handling from the biomarker working group for the mucopolysaccharidoses

Long-term galsulfase treatment associated with improved survival of patients with mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): 15 year follow-up from the survey study

Sub-analysis of long-term elosulfase alfa treatment outcomes in adults with Morquio syndrome type A

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study

The Morquio A Registry Study (MARS): Improving the understanding of Morquio syndrome type A

Keratan sulfate (KS) analysis in patients with Morquio syndrome type A: LC-MS/MS analysis of KS disaccharides demonstrates that urine is a better source than plasma to monitor dynamic change in KS

Galsulfase for mucopolysaccharidosis type VI: analysis of clinical data since 2000

Orphan drugs. BioMarin Europe replies

Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux–Lamy Syndrome)

BioMarin Europe replies

Cardiovascular Safety of the Oral Controlled Absorption System (OCAS) Formulation of Tamsulosin Compared to the Modified Release (MR) Formulation

Comparison of Vascular α1-Adrenoceptor Antagonism of Tamsulosin in Oral Controlled Absorption System (OCAS) and Modified Release (MR) Formulations

Inhibition of Graft Coronary Arteriosclerosis after Heart Transplantation

Comparison of Vascular α 1-Adrenoceptor Antagonism of Tamsulosin in Oral Controlled Absorption System (OCAS) and Modified Release (MR) Formulations

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