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Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what's new on the horizon?

Expert opinion on pharmacotherapy, 2024-07, Vol.25 (11), p.1-1438

2024

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RNAing toward a new therapy for hemophilia

Blood, 2024-05, Vol.143 (22), p.2219-2221

2024

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Unresolved hemostasis issues in haemophilia

Haemophilia : the official journal of the World Federation of Hemophilia, 2024-04, Vol.30 (S3), p.70-77

2024

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Health issues in women and girls affected by haemophilia with a focus on nomenclature, heavy menstrual bleeding, and musculoskeletal issues

Haemophilia : the official journal of the World Federation of Hemophilia, 2022-05, Vol.28 (S4), p.18-25

2022

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A cross‐sectional study of bleeding phenotype in haemophilia A carriers

British journal of haematology, 2015-07, Vol.170 (2), p.223-228

2015

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Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis

Haemophilia : the official journal of the World Federation of Hemophilia, 2019-09, Vol.25 (5), p.789-796

2019

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Pharmacokinetic parameters of recombinant factor IX Fc fusion protein are not influenced by factor IX antigen levels in subjects from the Phase 3 B‐LONG trial

Haemophilia : the official journal of the World Federation of Hemophilia, 2023-01, Vol.29 (1), p.404-407

2023

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Discussing investigational AAV gene therapy with hemophilia patients: A guide

Blood reviews, 2021-05, Vol.47, p.100759-100759, Article 100759

2021

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Hemophilia B (Factor IX Deficiency)

Hematology/oncology clinics of North America, 2021-12, Vol.35 (6), p.1143-1155

2021

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Assessment of menstrual health in adolescent and young adults with sickle cell disease

Pediatric blood & cancer, 2024-01, Vol.71 (1), p.e30727-n/a

2024

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Diagnostic pitfalls and conundrums in type 1 von Willebrand disease

Hematology, 2022-12, Vol.2022 (1), p.618-623

2022

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Substitution therapy

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-02, Vol.27 (S3), p.53-59

2021

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Severe muscle bleeds in children and young adults with hemophilia A on emicizumab prophylaxis: Real-world retrospective multi-institutional cohort

American journal of hematology, 2023-10, Vol.98 (10), p.E285-E287

2023

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Early diagnosis of persons with von Willebrand disease using a machine learning algorithm and real-world data

Expert review of hematology, 2024-06, Vol.17 (6), p.261-268

2024

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Thrombin generation and implications for hemophilia therapies: A narrative review

Research and practice in thrombosis and haemostasis, 2023-01, Vol.7 (1), p.100018-100018, Article 100018

2023

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Adequate menstrual suppression in adolescents with inherited bleeding disorders often requires multiple treatment changes: Retrospective cohort study of a multidisciplinary clinic

Pediatric blood & cancer, 2024-06, Vol.71 (6), p.e30944-n/a

2024

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Plasma factor IX: The tip of the iceberg?

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-05, Vol.27 (3), p.329-331

2021

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Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A

Haemophilia : the official journal of the World Federation of Hemophilia, 2024-07, Vol.30 (4), p.905-913

2024

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Conformational activation and inhibition of von Willebrand factor by targeting its autoinhibitory module

Blood, 2024-05, Vol.143 (19), p.1992-2004

2024

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for mucocutaneous bleeding disorders

Expert review of hematology, 2023-03, Vol.16 (sup1), p.39-54

2023

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Spontaneous bleeding and poor bleeding response with extended half‐life factor IX products: A survey of select US haemophilia treatment centres

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-05, Vol.26 (3), p.e128-e129

2020

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Dental habits and oral health in children and adolescents with bleeding disorders: A single‐institution cross‐sectional study

Haemophilia : the official journal of the World Federation of Hemophilia, 2022-01, Vol.28 (1), p.73-79

2022

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Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study

Orphanet journal of rare diseases, 2021-04, Vol.16 (1), p.189-189, Article 189

2021

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Depression and anxiety in persons with Von Willebrand disease

Haemophilia : the official journal of the World Federation of Hemophilia, 2023-03, Vol.29 (2), p.545-554

2023

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Novel therapeutics for hemophilia and other bleeding disorders

Blood, 2018-07, Vol.132 (1), p.23-30

2018

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Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what's new on the horizon?

RNAing toward a new therapy for hemophilia

Unresolved hemostasis issues in haemophilia

Health issues in women and girls affected by haemophilia with a focus on nomenclature, heavy menstrual bleeding, and musculoskeletal issues

A cross‐sectional study of bleeding phenotype in haemophilia A carriers

Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis

Pharmacokinetic parameters of recombinant factor IX Fc fusion protein are not influenced by factor IX antigen levels in subjects from the Phase 3 B‐LONG trial

Discussing investigational AAV gene therapy with hemophilia patients: A guide

Hemophilia B (Factor IX Deficiency)

Assessment of menstrual health in adolescent and young adults with sickle cell disease

Diagnostic pitfalls and conundrums in type 1 von Willebrand disease

Substitution therapy

Severe muscle bleeds in children and young adults with hemophilia A on emicizumab prophylaxis: Real-world retrospective multi-institutional cohort

Early diagnosis of persons with von Willebrand disease using a machine learning algorithm and real-world data

Thrombin generation and implications for hemophilia therapies: A narrative review

Adequate menstrual suppression in adolescents with inherited bleeding disorders often requires multiple treatment changes: Retrospective cohort study of a multidisciplinary clinic

Plasma factor IX: The tip of the iceberg?

Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A

Conformational activation and inhibition of von Willebrand factor by targeting its autoinhibitory module

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for mucocutaneous bleeding disorders

Spontaneous bleeding and poor bleeding response with extended half‐life factor IX products: A survey of select US haemophilia treatment centres

Dental habits and oral health in children and adolescents with bleeding disorders: A single‐institution cross‐sectional study

Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study

Depression and anxiety in persons with Von Willebrand disease

Novel therapeutics for hemophilia and other bleeding disorders

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