Last Posted: Aug 30, 2018
- Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.
Mahlangu Johnny et al. The New England journal of medicine 2018 Aug (9) 811-822 - Mimicking Factor VIII to Manage the Factor VIII-Deficient State.
Ragni Margaret V et al. The New England journal of medicine 2018 Aug (9) 880-882 - The Patients Who Don't Want to Be Cured A hemophiliac says his genetic disorder is part of his identity, and therapies like CRISPR threaten to erase it.
S Zhang, The Atlantic, August 29, 2018 - 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 - Consensus Statement of the Indian Academy of Pediatrics in Diagnosis and Management of Hemophilia.
Sachdeva Anupam et al. Indian pediatrics 2018 Jul 55(7) 582-590 - Effectiveness of a comprehensive educational programme for Accredited Social Health Activists (ASHAs) to identify individuals in the Udupi district with bleeding disorders: A community-based survey.
Badagabettu S et al. Haemophilia : the official journal of the World Federation of Hemophilia 2018 Aug - Factor IX Gene (F9) Genotyping Trends and Spectrum of Mutations Identified: A Reference Laboratory Experience.
Perez Botero Juliana et al. Seminars in thrombosis and hemostasis 2018 Apr 44(3) 287-292 - 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 Issues Set of Gene Therapy Draft Guidances
Global Genes, July 11, 2018
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