Last Posted: Aug 20, 2020
- Beliefs and Values About Gene Therapy and In-Utero Gene Editing in Patients with Hemophilia and Their Relatives.
Vasquez-Loarte Tania C et al. The patient 2020 Aug
- Update on Molecular Testing in von Willebrand Disease.
Batlle Javier et al. Seminars in thrombosis and hemostasis 2019 Apr
- Hemophilia carrier's awareness, diagnosis, and management in emerging countries: a cross-sectional study in Côte d'Ivoire (Ivory Coast).
Lambert Catherine et al. Orphanet journal of rare diseases 2019 Feb 14(1) 26
- Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.
Mahlangu Johnny et al. The New England journal of medicine 2018 Aug 379(9) 811-822
- Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A.
Shima Midori et al. The New England journal of medicine 2016 May 374(21) 2044-53
- They Thought Hemophilia Was a Lifelong Thing. They May Be Wrong.
Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients wary, worried that success will not last.
G Kolata, New York Times, August 13, 2018
- Gene therapy in hemophilia A: a cost-effectiveness analysis.
Machin Nicoletta et al. Blood advances 2018 Jul 2(14) 1792-1798
- FDA plans to speed path to approval for some gene therapies, starting with hemophilia
I. Swetlitz, StatNews, May 22, 2018
- Preconception Carrier Screening by Genome Sequencing: Results from the Clinical Laboratory.
Punj Sumit et al. American journal of human genetics 2018 May
- What is an inhibitor?
All people with hemophilia and VWD type 3 are at risk for developing an inhibitor ? an antibody ? to treatment used to stop or to prevent a bleeding episode: Anthony's story.
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