• "Liver-related aspects of gene therapy for hemophilia: need for collaborations with hepatologists": comment
• A Case of "Anaphylaxis" to NovoSeven in a Hemophiliac Patient
• A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B
• A Panoramic X-ray as a Supportive Diagnostic Tool for the Screening of Osteoporosis in Patients with Hemophilia A and B
• Augmented Degradation of Factors VIII and IX in the Intermittent Movement State
• Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from a Spanish reference centre
• Biodistribution of recombinant factor IX, extended half-life recombinant factor IX Fc fusion protein, and glycoPEGylated recombinant factor IX in hemophilia B mice
• Cellular stress and coagulation factor production: when more is not necessarily better
• Cellular stress and coagulation factor production: when more isn't necessarily better
• Characteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets
• Characterization of a recombinant factor IX molecule fused to coagulation factor XIII-B subunit
• Clinical course and prognosis of patients with hepatocellular carcinoma and haemophilia
• Clinical Implications of Discrepancy between One-stage Clotting and Chromogenic Factor IX Activity in Haemophilia B
• Clinical Profile of Adult Hemophilia Patients with Special Reference to FISH and WFHPE Score: An Observational Cross-sectional Study
• Clinicopathological Parameters of Haemophilia Patients at a Tertiary Care Centre in Northern India
• Complete F9 gene deletion, duplication and triplication rearrangements: implications for factor IX expression and clinical phenotypes
• Congenital Bleeding Disorders and COVID-19-A Systematic Literature Review
• Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK
• Efficacy of rFIXFc versus N9-GP Prophylaxis in Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PARADIGM 2 Trials
• Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy
• Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B
• Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency)
• Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia
• Evaluation of treatment and outcome for patients with haemophilia A and haemophilia B on extended half-life (EHL) factor products: A 12-month data analysis
• Evolution of Antidrug Antibody Assays During the Development of Anti-Tissue Factor Pathway Inhibitor Monoclonal Antibody Marstacimab
• Exploring factors impacting haplotype-based noninvasive prenatal diagnosis for single-gene recessive disorders
• Extended half-life factor IX prophylaxis up to every 21 days in hemophilia B: a longitudinal analysis of the efficacy and safety in selected adult patients
• Gene Therapy Approaches for the Treatment of Hemophilia B
• Genotype Hemophilia Screening Program Identified 2 Novel Variants Including a Novel Variant (c.5816-2A > G) Causing a Pathogenic Variant of the Factor 8 Gene
• Haemophilia B in Algeria: Realities and therapeutic perspectives
• Health-related quality of life and physical activity in Nordic patients with moderate haemophilia A and B (the MoHem study)
• Heart surgery in a pediatric patient with congenital heart disease and hemophilia B: A case report from Sudan
• Hemophilia B
• Hemophilia gene therapy: first, do no harm
• Hemophilia Gene Therapy: The End of the Beginning?
• Hemophilia patients: are they naturally anticoagulated?
• High variability in Factor IX one-stage assay in samples spiked with nonacog beta pegol among different pairs of reagent/detection system
• In vivo intranasal delivery of coagulation factor IX: a proof-of-concept study
• Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half-life factor IX therapies for severe or moderately severe haemophilia B
• Looking to the future of gene therapy for hemophilia A and B
• Managing Relevant Clinical Conditions of Hemophilia A/B Patients
• Model-Informed Support of Dose Selection for Prophylactic Treatment with Dalcinonacog Alfa in Adult and Paediatric Hemophilia B Patients
• Moderate hemophilia A and B patients with a severe bleeding phenotype have an increased burden of disease
• Molecular Evaluation and Vector Integration Analysis of HCC Complicating AAV Gene Therapy for Hemophilia B
• Neurological Complications Associated with Hereditary Bleeding Disorders
• Nonacog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Moderately Severe or Severe Hemophilia B in India
• Patients with moderate hemophilia A and B with a severe bleeding phenotype have an increased burden of disease
• Patients' and health care providers' perspectives on quality of hemophilia care in the Netherlands: a questionnaire and interview study
• Perioperative management of a patient with haemophilia B and PSVT undergoing radiofrequency ablation: A case report
• Pharmacokinetic-guided versus standard prophylaxis in hemophilia- A systematic review and meta-analysis
• Pharmacokinetic-guided versus standard prophylaxis in hemophilia: a systematic review and meta-analysis
• Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond
• Protein S Antibody as an Adjunct Therapy for Hemophilia B
• Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study
• Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries
• Recombinant factor IX Fc for major surgery in hemophilia B: factor IX plasma activity levels and effective hemostasis
• Switching and increasing prophylaxis regimen with a genetically recombinant fusion of coagulation factor IX and albumin in haemophilia B: a case report
• The frequency of complications in a cohort of patients diagnosed with hemophilia A and hemophilia B receiving prophylactic treatment in Colombia: A retrospective noninterventional study
• THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE
• Transition readiness among adolescents and young adults with haemophilia in the Netherlands: Nationwide questionnaire study
• Treatment of congenital coagulopathies, from biologic to biotechnological drugs: The relevance of gene editing (CRISPR/Cas)
• Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid
• Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B