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Use of plasma‐derived factor X concentrate in neonates and infants with congenital factor X deficiency
Journal of thrombosis and haemostasis, 2020-10, Vol.18 (10), p.2551-2556
2020
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Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Gene therapy, 2019-12, Vol.26 (12), p.504-514
2019
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The B‐Natural study—The outcome of immune tolerance induction therapy in patients with severe haemophilia B
Haemophilia : the official journal of the World Federation of Hemophilia, 2021-09, Vol.27 (5), p.802-813
2021
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Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity
Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.259-268
2020
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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs
Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.257-267
2018
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Natural history study of factor IX deficiency with focus on treatment and complications (B‐Natural)
Haemophilia : the official journal of the World Federation of Hemophilia, 2021-01, Vol.27 (1), p.49-59
2021
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Current practices in pediatric hospital‐acquired thromboembolism: Survey of the Children's Hospital Acquired Thrombosis (CHAT) Consortium
Research and practice in thrombosis and haemostasis, 2022-10, Vol.6 (7), p.e12793-e12793, Article e12793
2022
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Recombinant Porcine Factor VIII Use in Bleed Treatment in Non-Severe Hemophilia a Inhibitor Patients: Dosing Strategies and Efficacy
Blood, 2018-11, Vol.132 (Supplement 1), p.1202-1202
2018
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Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors
Blood, 2022-11, Vol.140 (Supplement 1), p.8485-8488
2022
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Recombinant porcine factor VIII use in bleed treatment in non‐severe haemophilia A inhibitor patients: Dosing strategies and efficacy
Haemophilia : the official journal of the World Federation of Hemophilia, 2021-01, Vol.27 (1), p.e147-e150
2021
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Excessive breakthrough bleeding in haemophilia B patients on factor IX‐albumin fusion protein prophylactic therapy: A single centre case series
Haemophilia : the official journal of the World Federation of Hemophilia, 2020-01, Vol.26 (1), p.e23-e25
2020
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Immune Tolerance Induction for FIX Inhibitors Using Combined B and T Cell Immune Modulation Therapy in Severe Hemophilia B
BLOOD, 2018-11, Vol.132 (Supplement 1), p.1193-1193
2018
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The predictive value of factor VIII/factor IX levels to define the severity of hemophilia: communication from the SSC of ISTH
Journal of thrombosis and haemostasis, 2018-10, Vol.16 (10), p.2106-2110
2018
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Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR)
Blood cells, molecules, & diseases, 2021-03, Vol.87, p.102534-102534, Article 102534
2021
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HTRS2023.O8C.2 Eptacog beta efficacy and safety in underweight, normal weight, and overweight/obese persons with hemophilia A or B and inhibitors
Research and practice in thrombosis and haemostasis, 2023-08, Vol.7, p.100219, Article 100219
2023
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Recombinant porcine factor VIII for high risk surgery in pediatric congenital hemophilia A with high-titer inhibitor
Haemophilia : the official journal of the World Federation of Hemophilia, 2017-01, Vol.23 (2), p.e93-e98
2017
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Gene Therapy for Bleeding Disorders
Nonmalignant Hematology, p.321-336
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