Two gene therapies for hemophilia available: Now what?
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference13 articles.
1. Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A;Mahlangu;N. Engl. J. Med.,2023
2. Gene therapy with etranacogene dezaparvovec for hemophilia B;Pipe;N. Engl. J. Med.,2023
3. WFH Guidelines for the Management of Hemophilia;Srivastava;Haemophilia,2020
4. Efanesoctocog alfa prophylaxis for patients with severe hemophilia A;von Drygalski;N. Engl. J. Med.,2023
5. AAV integration in human hepatocytes;Dalwadi;Mol. Ther.,2021
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3. Development of the coreHEM mental health patient‐reported outcome measure – A novel mental health outlook measure for people with haemophilia;Haemophilia;2024-09-05
4. Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy;Journal of Thrombosis and Haemostasis;2024-05
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