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Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

Orphanet journal of rare diseases, 2021-10, Vol.16 (1), p.456-14, Article 456

2021

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Lipid‐based strategies used to identify extracellular vesicles in flow cytometry can be confounded by lipoproteins: Evaluations of annexin V, lactadherin, and detergent lysis

Journal of extracellular vesicles, 2022-04, Vol.11 (4), p.e12200-n/a

2022

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Conventional, High-Resolution and Imaging Flow Cytometry: Benchmarking Performance in Characterisation of Extracellular Vesicles

Biomedicines, 2021-01, Vol.9 (2), p.124

2021

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Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Molecular genetics and metabolism reports, 2023-12, Vol.37, p.101005-101005, Article 101005

2023

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Valsartan Improves Arterial Stiffness in Type 2 Diabetes Independently of Blood Pressure Lowering

Hypertension (Dallas, Tex. 1979), 2008-06, Vol.51 (6), p.1617-1623

2008

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Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry

Orphanet journal of rare diseases, 2022-06, Vol.17 (1), p.1-238, Article 238

2022

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Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study

Therapeutic advances in hematology, 2023-01, Vol.14, p.20406207231184323-20406207231184323

2023

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Bariatric surgery reduces CD36-bearing microvesicles of endothelial and monocyte origin

Nutrition & metabolism, 2018-10, Vol.15 (1), p.76-76, Article 76

2018

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Rationale and design of the multicentre, randomized, double-blind, placebo-controlled Aliskiren Trial on Acute Heart Failure Outcomes (ASTRONAUT)

European journal of heart failure, 2011-01, Vol.13 (1), p.100-106

2011

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Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry

Journal of clinical medicine, 2024-05, Vol.13 (10), p.2782

2024

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Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)

Journal of clinical medicine, 2022-08, Vol.11 (17), p.5158

2022

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Improvement in diagnostic delays over time in patients with hereditary angioedema: findings from the Icatibant Outcome Survey

Clinical and translational allergy, 2018-10, Vol.8 (1), p.42-n/a, Article 42

2018

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Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11 , 5158"

Journal of clinical medicine, 2023-06, Vol.12 (12), p.4017

2023

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Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease

Journal of clinical medicine, 2022-08, Vol.11 (16), p.4810

2022

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FindZebra online search delving into rare disease case reports using natural language processing

PLOS digital health, 2023-06, Vol.2 (6), p.e0000269-e0000269

2023

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Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry

Journal of clinical medicine, 2024-06, Vol.13 (12), p.3588

2024

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Clinical Profile Among Brazilian Mucopolysaccharidosis type II Patients: Subgroup Analysis from the Hunter Outcome Survey

Journal of inborn errors of metabolism and screening, 2023, Vol.11

2023

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Hereditary angioedema due to C1 inhibitor deficiency: real-world experience from the Icatibant Outcome Survey in Spain

Allergy, asthma, and clinical immunology, 2021-12, Vol.17 (1), p.137-137, Article 137

2021

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Zoom in on Antibody Aggregates: A Potential Pitfall in the Search of Rare EV Populations

Biomedicines, 2021-02, Vol.9 (2), p.206

2021

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Justice and the Missional Framework Document of the Dutch Reformed Church

Verbum et Ecclesia, 2017, Vol.38 (1), p.1-9

2017

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BLTR1 and CD36 Expressing Microvesicles in Atherosclerotic Patients and Healthy Individuals

Frontiers in cardiovascular medicine, 2018-10, Vol.5, p.156-156

2018

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Clinical outcomes in elderly patients receiving agalsidase alfa treatment in the Fabry Outcome Survey

Molecular genetics and metabolism, 2024-08, p.108561, Article 108561

2024

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Microvesicles Correlated with Components of Metabolic Syndrome in Men with Type 2 Diabetes Mellitus and Lowered Testosterone Levels But Were Unaltered by Testosterone Therapy

Journal of Diabetes Research, 2017-01, Vol.2017, p.4257875-9

2017

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Long-term treatment of Gaucher disease with velaglucerase alfa in a cohort of patients from Paraguay

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S70-S71

2021

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Orthopedic manifestations in patients with mucopolysaccharidosis type II enrolled in the Hunter Outcome Survey

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S65-S65

2021

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Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

Lipid‐based strategies used to identify extracellular vesicles in flow cytometry can be confounded by lipoproteins: Evaluations of annexin V, lactadherin, and detergent lysis

Conventional, High-Resolution and Imaging Flow Cytometry: Benchmarking Performance in Characterisation of Extracellular Vesicles

Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Valsartan Improves Arterial Stiffness in Type 2 Diabetes Independently of Blood Pressure Lowering

Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry

Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study

Bariatric surgery reduces CD36-bearing microvesicles of endothelial and monocyte origin

Rationale and design of the multicentre, randomized, double-blind, placebo-controlled Aliskiren Trial on Acute Heart Failure Outcomes (ASTRONAUT)

Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry

Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)

Improvement in diagnostic delays over time in patients with hereditary angioedema: findings from the Icatibant Outcome Survey

Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease

FindZebra online search delving into rare disease case reports using natural language processing

Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry

Clinical Profile Among Brazilian Mucopolysaccharidosis type II Patients: Subgroup Analysis from the Hunter Outcome Survey

Hereditary angioedema due to C1 inhibitor deficiency: real-world experience from the Icatibant Outcome Survey in Spain

Zoom in on Antibody Aggregates: A Potential Pitfall in the Search of Rare EV Populations

Justice and the Missional Framework Document of the Dutch Reformed Church

BLTR1 and CD36 Expressing Microvesicles in Atherosclerotic Patients and Healthy Individuals

Clinical outcomes in elderly patients receiving agalsidase alfa treatment in the Fabry Outcome Survey

Microvesicles Correlated with Components of Metabolic Syndrome in Men with Type 2 Diabetes Mellitus and Lowered Testosterone Levels But Were Unaltered by Testosterone Therapy

Long-term treatment of Gaucher disease with velaglucerase alfa in a cohort of patients from Paraguay

Orthopedic manifestations in patients with mucopolysaccharidosis type II enrolled in the Hunter Outcome Survey

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