专题：Hemophilia Treatment and Research

This cluster of papers focuses on the management and treatment of hemophilia, including prophylaxis, inhibitors, gene therapy, and the impact on joint health. It covers a wide range of topics such as recombinant factor VIII, factor IX, and the safety and efficacy of various treatment strategies.
最新文献
Real-world quality of life in patients with hereditary angioedema receiving lanadelumab or other long-term prophylaxis

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Immune Response Mechanisms in Haemophilia A

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Structural Analysis of von Willebrand Factor

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Gene Therapy of Haemophilia: Current Status and Future Directions

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Thrombin Generation Assays: Possibilities and Limitations

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Parental experience of hemophilia diagnosis disclosure in Morocco: a mixed-methods study on communication quality and psychosocial impact

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JAMA Pediatrics

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Global prevalence of hereditary hemorrhagic telangiectasia-associated variants estimated by analysis of large-scale genomic databases

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Infigratinib is a weak inhibitor of the FGFR3-N540K mutant associated with hypochondroplasia

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Updated global prevalence and ethnic diversity of von Willebrand disease based on population genetics analysis

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近5年高被引文献
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

article Full Text OpenAlex 365 FWCI56.135

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

article Full Text OpenAlex 312 FWCI92.3042

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

article Full Text OpenAlex 177 FWCI52.3723

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A

article Full Text OpenAlex 161 FWCI47.0463

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

article Full Text OpenAlex 145 FWCI26.349

Thrombophilia testing: A British Society for Haematology guideline

letter Full Text OpenAlex 124 FWCI18.2834

Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial

article Full Text OpenAlex 108 FWCI31.3159

The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study

article Full Text OpenAlex 103 FWCI15.7172

Gene therapy for hemophilia

review Full Text OpenAlex 100 FWCI17.765

Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A

article Full Text OpenAlex 97 FWCI17.1839