Impact of Emicizumab on Hemophilia Management
Author:
Affiliation:
1. Department of Pediatrics and Child Health, Nihon University School of Medicine
Publisher
The Nihon University Medical Association
Subject
Applied Mathematics,General Mathematics
Link
https://www.jstage.jst.go.jp/article/numa/82/1/82_23/_pdf
Reference18 articles.
1. 1) Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535–544.
2. 2) Konkle BA, Walsh CE, Escobar MA, et al. BAX 335 hemophilia B gene therapy clinical trial results: Potential impact of CpG sequences on gene expression. Blood 2021; 137: 763– 774.
3. 3) Rosen S, Tiefenbacher S, Robinson M, et al. Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy. Blood 2020; 136: 2524–2534.
4. 4) Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N Engl J Med 2020; 382: 29–40.
5. 5) Kitazawa T, Igawa T, Sampei Z, et al. A bispecific antibody to factor IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med 2012; 18: 1570–1574.
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