Progress, and prospects in the therapeutic armamentarium of persons with congenital hemophilia. Defining the place for liver-directed gene therapy
Author:
Publisher
Elsevier BV
Subject
Oncology,Hematology
Reference183 articles.
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1. The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art;Bleeding, Thrombosis and Vascular Biology;2024-09-04
2. Patient Perspective on Disease Burden and Gene Therapy for Hemophilia A and B: The “Haemvolution for Patients” Italian Survey;Seminars in Thrombosis and Hemostasis;2024-06-18
3. Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline;Blood Reviews;2023-11
4. Residual burden of liver disease after HCV clearance in hemophilia: a word of caution in the era of gene therapy;Blood Advances;2023-09-29
5. New directions to develop therapies for people with hemophilia;Expert Review of Hematology;2023-03-08
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