Hemophilia A
What's New
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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Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
Content Summary
- CDC Information (18)
- NIH Information (8)
- COVID-19 (3)
- CDC Publications (3)
- Human Genome Epidemiologic Studies (65)
- Genomic Tests Evidence Synthesis (3)
- Genomic Tests Guidelines (1)
- Tier-Classified Guidelines (3)
- Pathogen Advanced Molecular Detection (1)
- State Public Health Genomics Programs (7)
- Reviews/Commentaries (13)
- Tools/Methods (1)
- Ethical/Legal and Social Issues (ELSI) (1)
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Eclampsia
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Glomerulonephritis
- Graves Disease
- Hemophilia
- Huntington Disease
- Microcephaly
- Myasthenia Gravis
- Phenylketonuria
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
Disclaimer: Articles listed in the Public Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
- Page last reviewed:Oct 1, 2020
- Page last updated:Jan 21, 2021
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