Disease: Hemophilia B
- A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR
- A Panoramic X-ray as a Supportive Diagnostic Tool for the Screening of Osteoporosis in Patients with Hemophilia A and B
- 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
- Adherence tool for prophylactic haemophilia treatment in adult and adolescent patients: A systematic review and meta-analysis protocol
- Advances in AAV-CRISPR/Cas9-Mediated Hemophilia A Gene Therapy --Review
- Arthropathy on X-rays in 363 persons with hemophilia: long-term development, and impact of birth cohort and inhibitor status
- Assessing health plan payer's budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States
- Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study
- Breaking ground in haemophilia B gene therapy: insights from the HOPE-B trial and beyond
- Cellular stress and coagulation factor production: when more is not necessarily better
- Cellular stress and coagulation factor production: when more isn't necessarily better
- 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
- Characteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets
- Clinical Analysis and Mental Health Survey of Hemophilia Carriers: a Cross-sectional Study
- Clinical characteristic and management of haemophilia patients in Malaysia: A single centre experience
- Complete F9 gene deletion, duplication and triplication rearrangements: implications for factor IX expression and clinical phenotypes
- Congenital Haemostasis Disorders and Urology Surgery: Is It Safe?
- 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
- Current status of treatment and disease burden of a cohort of hemophilia B in China
- Deciphering conundrums of AAV liver-directed gene therapy: focus on hemophilia
- Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
- Disease Burden, Clinical Outcomes, and Quality of Life in People with Hemophilia A without Inhibitors in Europe: Analyses from CHESS II/CHESS PAEDs
- Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK
- 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
- Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis
- 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
- Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective
- Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B
- 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
- Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency)
- Exploring factors impacting haplotype-based noninvasive prenatal diagnosis for single-gene recessive disorders
- Extravascular factor IX pool fed by prophylaxis is a true hemostatic barrier against bleeding
- Factor IX administration in the skin primes inhibitor formation and sensitizes hemophilia B mice to systemic factor IX administration
- Fidanacogene Elaparvovec: First Approval
- Force-sensing treadmill gait analysis system can detect gait abnormalities in haemophilia patients without arthropathy
- Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review
- Genetic variant detection in a South African haemophilia B population
- 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?
- Health-related quality of life and physical activity in Nordic patients with moderate haemophilia A and B (the MoHem study)
- Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
- Humanization and functional characterization of enhanced coagulation factor IX variants identified through ancestral sequence reconstruction
- In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia
- Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half-life factor IX therapies for severe or moderately severe haemophilia B
- Inhibitor development according to concentrate in severe hemophilia: reporting on 1392 Previously Untreated Patients from Europe and Canada
- Inhibitors in hemophilia: association with surgery plans and outcomes in a retrospective cohort study
- Knowledge and attitudes toward gene therapy of a cohort of Italian patients with hemophilia
- KNOWLEDGE AND ATTITUDES TOWARDS GENE THERAPY OF A COHORT OF ITALIAN PATIENTS WITH HAEMOPHILIA
- Looking to the future of gene therapy for hemophilia A and B
- Management strategies in persons with inherited haemophilia requiring antithrombotic therapy: A scoping review
- Matching-Adjusted Indirect Comparison of Recombinant Factor IX Albumin Fusion Protein Versus Recombinant Factor IX Fc Fusion Protein for Weekly Prophylactic Treatment of Hemophilia B
- Moderate hemophilia A and B patients with a severe bleeding phenotype have an increased burden of disease
- Neurological Complications Associated with Hereditary Bleeding Disorders
- New perspective of anticoagulation in intensive care unit: basic and clinical advances in coagulation factor XII and XI inhibitors
- Nonacog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Moderately Severe or Severe Hemophilia B in India
- 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
- Observational cohort study of long-term outcomes of liver transplantation in haemophilia
- Origin of pathogenic variant and mosaicism in families with a sporadic case of haemophilia B
- Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey
- Patients with moderate hemophilia A and B with a severe bleeding phenotype have an increased burden of disease
- Pattern of use and clinical outcomes with rIX-FP in pediatric/adolescent patients with haemophilia B in Italy: Results from IDEAL real-world study
- Perioperative management of a patient with haemophilia B and PSVT undergoing radiofrequency ablation: A case report
- Perspectives and perception of haemophilia gene therapy by French patients
- Pleiotropic effects of different exonic nucleotide changes at the same position contribute to hemophilia B phenotypic variation
- Pleiotropic Effects of Different Exonic Nucleotide Changes at the Same Position Contributing to Hemophilia B Phenotypic Variation
- Portal vein thrombosis in a patient with severe hemophilia B: A challenging balanced management
- Predictive performance of pharmacokinetic-guided prophylactic dosing of factor concentrates in hemophilia A and B
- Prophylactic Treatment of Children with Hemophilia in Sweden
- Quality improvement in paediatric preoperative screening: a Japanese perspective
- Quantitative transcriptome-based analysis predicts a combination therapy for severe haemophilia B: A case report
- Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B
- Real-world experience of rIX-FP prophylaxis at dosing intervals of up to 14 days in a pediatric patient with hemophilia B during the COVID-19 pandemic
- Recent advances in the replacement therapy for Hemophilia
- Recombinant factor IX Fc for the treatment of hemophilia B
- Relationship between plasma tissue Factor Pathway Inhibitor (TFPI) levels, thrombin generation and clinical risk of bleeding in patients with severe haemophilia A or B
- 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
- Skeletal complications in patients with hemophilia: a single-center experience
- Soluble expression of recombinant coagulation factor IX protein using <em>Escherichia coli</em>
- Subcutaneous Marzeptacog Alfa (Activated) for On-Demand Treatment of Bleeding Events in Subjects With Hemophilia A or B With Inhibitors
- Suspected autoimmune coagulation factor IX deficiency difficult to diagnose due to concurrent lupus anticoagulant
- 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 current challenges faced by people with hemophilia B
- 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 good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment
- Treatment of congenital coagulopathies, from biologic to biotechnological drugs: The relevance of gene editing (CRISPR/Cas)
- Use of crushed tranexamic acid tablets in water for paediatric patients with bleeding disorders
- Use of nonacog beta pegol during surgery in persons with hemophilia B: a case series
- 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
- Variant spectrum of <em>F8</em> and <em>F9</em> in hemophilia patients from southern China and 26 novel variants
- Viral Vectors in Gene Replacement Therapy