• A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR
• A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B
• AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life
• AAV mediated gene therapy for haemophilia B: From the early attempts to modern trials
• AAV mediated genome engineering with a bypass coagulation factor alleviates the bleeding phenotype in a murine model of hemophilia B
• AAV vectors for long-term gene therapy of hemophilia B: Are we there yet?
• Altered Mental Status in a 19-year-old Male with Hemophilia B
• An integrated multi-tool analysis contributes elements to interpreting unclassified factor IX missense variants associated with haemophilia B
• An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B
• Analysis of long-term clinical and cost impact of etranacogene dezaparvovec for the treatment of hemophilia B population in the United States
• Arthropathy on X-rays in 363 persons with hemophilia: long-term development, and impact of birth cohort and inhibitor status
• Assessing health care resource use, outcomes, and costs among Medicaid beneficiaries receiving factor IX prophylaxis for hemophilia B
• Assessing health plan payer's budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States
• Beqvez - another gene therapy for hemophilia B
• Bioclinical features of haemophilia patients in Benin in 2023: Towards better care
• Bleeding phenotype according to factor level in 825 children with nonsevere hemophilia: data from the PedNet cohort
• Bleeds and resource use in hemophilia B: retrospective observational study
• Blood coagulation factor IX: structural insights impacting hemophilia B therapy
• Breaking ground in haemophilia B gene therapy: insights from the HOPE-B trial and beyond
• Cerebral infarction in a patient with inhibitor-positive hemophilia B treated with plasma-derived factor VIIa and factor X mixture
• Chain-mediating effects of kinesiophobia and self-efficacy on pain catastrophizing and physical activity in haemophilia patients
• Characterization of recombinant Factor IX fusion proteins enabling subcutaneous administration
• Clinical Analysis and Mental Health Survey of Hemophilia Carriers: a Cross-sectional Study
• Clinical analysis and quality of life survey of hemophilia B patients in the central and western regions of China
• Clinical characteristic and management of haemophilia patients in Malaysia: A single centre experience
• Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea
• Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies
• Comparative analysis of thrombin generation platforms for patients with coagulation factor deficiencies: A comprehensive assessment
• Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study
• Concurrent congenital hemophilia B and acquired hemophilia A: a unique case report
• Congenital Haemostasis Disorders and Urology Surgery: Is It Safe?
• Corrigendum to "Long-term correction of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration" [Journal of Genetics and Genomics (2022) 49, 1114-1126]
• Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany
• Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors
• Current and emerging Gene Therapies for Haemophilia A and B
• Disease and treatment burden of patients with haemophilia entering the explorer6 non-interventional study
• Disease Burden, Clinical Outcomes, and Quality of Life in People with Hemophilia A without Inhibitors in Europe: Analyses from CHESS II/CHESS PAEDs
• Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study
• Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy
• Enhanced Procoagulant Activity of Select Hemophilia B Causing Factor IX Variants with Emicizumab
• Epidemiological profile of hemophilia A in Karbala-Iraq
• Epidemiology, patient journey and unmet needs related to hemophilia in Brazil: a scoping review with evidence map
• Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
• Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment
• Fidanacogene Elaparvovec: First Approval
• Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec
• Force-sensing treadmill gait analysis system can detect gait abnormalities in haemophilia patients without arthropathy
• Gene Therapy for Hemophilia B: Achievements, Open Issues, and Perspectives
• Gene therapy for people with Haemophilia B: a proposed care delivery model in Italy
• Genetic variant detection in a South African haemophilia B population
• Genotype-phenotype analyses of Iranian patients with hemophilia B (Leyden -) and hemophilia B (Leyden +): A single-center study
• Haemophilia B therapy: impact of the reimbursability of a long-acting recombinant factor on pharmaceutical expenditure in Italy
• Haemophilia care in Asia: Learning from clinical practice in some Asian countries
• Haemophilia in the era of novel therapies: Where do inhibitors feature in the new landscape?
• Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
• How clinicians and persons with hemophilia may approach shared decision-making
• Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
• Identifying hemophilia B carriers: Utility of aPTT, factor IX levels and ratios of factor IX to other Vitamin K dependent factors
• In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia
• Inhibitor development according to concentrate after 50 exposure days in severe hemophilia: data from the European HAemophilia Safety Surveillance (EUHASS)
• International Society on Thrombosis and Haemostasis clinical practice guideline for treatment of congenital hemophilia A and B based on the Grading of Recommendations Assessment, Development, and Evaluation methodology
• Long-term Clinical Outcomes of Prophylaxis With a rFVIIIFc or rFIXFc in Adults 50+ Years of Age With Hemophilia A or B
• Matched cohort study evaluating the hemostatic efficacy of fibrin sealant versus conventional approaches following dental surgery in patients with hemophilia
• Minimal interference of concizumab with standard clinical coagulation laboratory assays - An in vitro study
• Multimodal mechanisms of pathogenic variants in the signal peptide of FIX leading to hemophilia B
• Multiyear, real-world, retrospective cohort study using a patient-centric approach to evaluate the burden of haemophilia B in the United States
• Navigating the Challenges of Factor X Deficiency: A Case Study
• New perspective of anticoagulation in intensive care unit: basic and clinical advances in coagulation factor XII and XI inhibitors
• NICE recommends new gene therapy for adults with haemophilia B
• Nonacog beta pegol prophylaxis in children with hemophilia B: safety, efficacy, and neurodevelopmental outcomes for up to 8 years
• Noninvasive twin genotyping for recessive monogenic disorders by relative haplotype dosage
• Origin of pathogenic variant and mosaicism in families with a sporadic case of haemophilia B
• Patient Perspective on Disease Burden and Gene Therapy for Hemophilia A and B: The "Haemvolution for Patients" Italian Survey
• Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey
• Predictive performance of pharmacokinetic-guided prophylactic dosing of factor concentrates in hemophilia A and B
• Primary postpartum hemorrhage in women with von Willebrand disease and carriers of hemophilia: a retrospective analysis
• Prophylactic Treatment of Children with Hemophilia in Sweden
• Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B-LONG study
• Real-world experience of rIX-FP prophylaxis at dosing intervals of 14 days or more in adult patients with haemophilia B in Italy - Results from IDEAL Part B
• Real-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany
• Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives
• Recent advances in the replacement therapy for Hemophilia
• Recombinant factor IX Fc for the treatment of hemophilia B
• Regular prophylaxis with activated prothrombin complex concentrates in pediatric hemophilia
• Relationship between plasma tissue Factor Pathway Inhibitor (TFPI) levels, thrombin generation and clinical risk of bleeding in patients with severe haemophilia A or B
• Roctavian Gene Therapy for Hemophilia A
• Safety and Efficacy of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Untreated Patients with Hemophilia B
• Six-Year, Real-World Use of Prophylaxis with Recombinant Factor IX-Albumin Fusion Protein (rIX-FP) in Persons with Hemophilia B: A Single-Center Retrospective-Prospective Study
• Soluble expression of recombinant coagulation factor IX protein using <em>Escherichia coli</em>
• Spontaneous resolution and bone regeneration in hemophilic pseudotumor: A rare case report and literature review
• Study on the Mutation of FⅨ Gene in 31 Patients with Type B Hemophilia
• Successful immunosuppressive drug-free immune tolerance induction in hemophilia B with inhibitor and anaphylaxis to factor IX: A case report
• The benefits of gene therapy in people with haemophilia
• The complex, confusing and poorly understood immune responses to AAV-mediated gene transfer in haemophilia-Is more or less immunosuppression required?
• The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference
• The good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment
• The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B
• TKR in Hemophilic Arthropathy: A Combination of Special Surgical Considerations and Novel Nonacog Beta Pegol: A Case Report
• Use of crushed tranexamic acid tablets in water for paediatric patients with bleeding disorders
• Viral Vectors in Gene Replacement Therapy