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BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A

The New England journal of medicine, 2020-09, Vol.383 (11), p.1018-1027

2020

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Shortened Lifespan and Lethal Hemorrhage in a Hemophilia A Mouse Model

PloS one, 2016-05, Vol.11 (5), p.e0154857-e0154857

2016

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ADAMTS13 Retards Progression of Diabetic Nephropathy by Inhibiting Intrarenal Thrombosis in Mice

Arteriosclerosis, thrombosis, and vascular biology, 2017-07, Vol.37 (7), p.1332-1338

2017

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The intersection of vector biology, gene therapy, and hemophilia

Research and practice in thrombosis and haemostasis, 2021-08, Vol.5 (6), p.e12586-n/a, Article e12586

2021

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Haemostatic efficacy of single‐dose factor administration in neonates with severe haemophilia undergoing circumcision

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-09, Vol.26 (5), p.786-792

2020

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Successful Management of Blue Rubber Bleb Nevus Syndrome (BRBNS) with Sirolimus

Case reports in pediatrics, 2018-01, Vol.2018, p.7654278-5

2018

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A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon

Molecular therapy. Nucleic acids, 2012-10, Vol.1 (10), p.e50-e50, Article e50

2012

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Endothelial Cell-Derived von Willebrand Factor Is the Major Determinant That Mediates von Willebrand Factor-Dependent Acute Ischemic Stroke by Promoting Postischemic Thrombo-Inflammation

Arteriosclerosis, thrombosis, and vascular biology, 2016-09, Vol.36 (9), p.1829-1837

2016

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Endothelial Cell-Derived Von Willebrand Factor, But Not Platelet-Derived, Promotes Atherosclerosis in Apolipoprotein E-Deficient Mice

Arteriosclerosis, thrombosis, and vascular biology, 2018-03, Vol.38 (3), p.520-528

2018

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Self-concept and academic achievement in children with chronic kidney disease

Pediatric nephrology (Berlin, West), 2024-03, Vol.39 (3), p.819-827

2024

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Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A

The New England journal of medicine, 2024-07, Vol.391 (3), p.235-246

2024

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Trends in dedicated care for females with bleeding disorders within U.S. hemophilia treatment centers

American journal of hematology, 2023-12, Vol.98 (12), p.E399-E402

2023

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Mental health disorders in haemophilia: Systematic literature review and meta‐analysis

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-05, Vol.26 (3), p.431-442

2020

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Laboratory misdiagnosis of von Willebrand disease in post‐menarchal females: A multi‐center study

American journal of hematology, 2020-09, Vol.95 (9), p.1022-1029

2020

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Real‐world data of immune tolerance induction using recombinant factor VIII Fc fusion protein in patients with severe haemophilia A with inhibitors at high risk for immune tolerance induction failure: A follow‐up retrospective analysis

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-01, Vol.27 (1), p.19-25

2021

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Respiratory Syncytial Virus Infection Results in Activation of Multiple Protein Kinase C Isoforms Leading to Activation of Mitogen-Activated Protein Kinase

The Journal of immunology (1950), 2001-02, Vol.166 (4), p.2681-2687

2001

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piggyBac-mediated phenotypic correction of factor VIII deficiency

Molecular therapy. Methods & clinical development, 2014-01, Vol.1 (C), p.14042-14042, Article 14042

2014

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Respiratory Syncytial Virus Inhibits Apoptosis and Induces NF-κB Activity through a Phosphatidylinositol 3-Kinase-dependent Pathway

The Journal of biological chemistry, 2002-01, Vol.277 (1), p.492-501

2002

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Longitudinal Efficacy and Safety of N8-GP for Prophylaxis and Bleed Control in US Patients

Blood, 2021-11, Vol.138 (Supplement 1), p.3205-3205

2021

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Brain Anomalies in Children with Severe Factor VIII Deficiency- a Pilot Study

Blood, 2019-11, Vol.134 (Supplement_1), p.1121-1121

2019

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PREDICT: A Multicenter, Prospective, Open-Label, Clinical Study Using a New Risk Score Approach to Assess the Most Appropriate Prophylaxis Regimen to Reach Favorable Outcomes in Hemophilia A, When Switching from Standard-Half-Life Products to Damoctocog Alfa Pegol

Blood, 2021-11, Vol.138 (Supplement 1), p.2115-2115

2021

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Long-Term Expression of Von Willebrand Factor Via Piggybac-Mediated Gene Transfer

Blood, 2016-12, Vol.128 (22), p.3511-3511

2016

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Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

Blood, 2021-11, Vol.138 (Supplement 1), p.1035-1035

2021

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The Discordance between Offsite to Onsite Testing for Von Willebrand Disease in Post-Menarchal Females: A Multi-Center Study

Blood, 2019-11, Vol.134 (Supplement_1), p.628-628

2019

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Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B

Blood, 2020-11, Vol.136 (Supplement 1), p.2-3

2020

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BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A

Shortened Lifespan and Lethal Hemorrhage in a Hemophilia A Mouse Model

ADAMTS13 Retards Progression of Diabetic Nephropathy by Inhibiting Intrarenal Thrombosis in Mice

The intersection of vector biology, gene therapy, and hemophilia

Haemostatic efficacy of single‐dose factor administration in neonates with severe haemophilia undergoing circumcision

Successful Management of Blue Rubber Bleb Nevus Syndrome (BRBNS) with Sirolimus

A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon

Endothelial Cell-Derived von Willebrand Factor Is the Major Determinant That Mediates von Willebrand Factor-Dependent Acute Ischemic Stroke by Promoting Postischemic Thrombo-Inflammation

Endothelial Cell-Derived Von Willebrand Factor, But Not Platelet-Derived, Promotes Atherosclerosis in Apolipoprotein E-Deficient Mice

Self-concept and academic achievement in children with chronic kidney disease

Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A

Trends in dedicated care for females with bleeding disorders within U.S. hemophilia treatment centers

Mental health disorders in haemophilia: Systematic literature review and meta‐analysis

Laboratory misdiagnosis of von Willebrand disease in post‐menarchal females: A multi‐center study

Real‐world data of immune tolerance induction using recombinant factor VIII Fc fusion protein in patients with severe haemophilia A with inhibitors at high risk for immune tolerance induction failure: A follow‐up retrospective analysis

Respiratory Syncytial Virus Infection Results in Activation of Multiple Protein Kinase C Isoforms Leading to Activation of Mitogen-Activated Protein Kinase

piggyBac-mediated phenotypic correction of factor VIII deficiency

Respiratory Syncytial Virus Inhibits Apoptosis and Induces NF-κB Activity through a Phosphatidylinositol 3-Kinase-dependent Pathway

Longitudinal Efficacy and Safety of N8-GP for Prophylaxis and Bleed Control in US Patients

Brain Anomalies in Children with Severe Factor VIII Deficiency- a Pilot Study

PREDICT: A Multicenter, Prospective, Open-Label, Clinical Study Using a New Risk Score Approach to Assess the Most Appropriate Prophylaxis Regimen to Reach Favorable Outcomes in Hemophilia A, When Switching from Standard-Half-Life Products to Damoctocog Alfa Pegol

Long-Term Expression of Von Willebrand Factor Via Piggybac-Mediated Gene Transfer

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

The Discordance between Offsite to Onsite Testing for Von Willebrand Disease in Post-Menarchal Females: A Multi-Center Study

Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B

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