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.
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The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

article Full Text OpenAlex 536 FWCI51.3392

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

article Full Text OpenAlex 429 FWCI55.5873

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

article Full Text OpenAlex 365 FWCI64.4361

Thalassaemia

article Full Text OpenAlex 314 FWCI29.9227

2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia

article Full Text OpenAlex 311 FWCI28.1421

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A

article Full Text OpenAlex 208 FWCI36.3667

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

article Full Text OpenAlex 206 FWCI36.3667

CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema

article Full Text OpenAlex 192 FWCI44.0388

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

article Full Text OpenAlex 191 FWCI25.0689

Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission

article Full Text OpenAlex 181 FWCI25.5405