Last Posted: Feb 12, 2020
- Investigational Hemophilia A Gene Therapy Shows Sustained Benefit
J Abbasi, JAMA, February 11, 2020
- Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries.
Iorio Alfonso et al. Annals of internal medicine 2019 Sep
- Mothers' intentions and behaviours regarding providing risk communication to their daughters about their possibility of being haemophilia carriers: A qualitative study.
Fujii Tomie et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Oct
- Was the Human Genome Project a dud?
EF Torrey, Dallas Morning News, October 13, 2019
- Establishment of diagnostic facilities for autosomal recessive bleeding disorders in Pakistan.
Naz Arshi et al. Blood advances 2018 2(Suppl 1) 35-38
- Management of haemophilia carriers around the time of their delivery: Phenotypic variation requiring customization of management.
Girgis M R et al. Haemophilia : the official journal of the World Federation of Hemophilia 2018 24(3) e128-e129
- Gene therapy for hemophilia: Progress to date and challenges moving forward.
Gollomp Kandace L et al. Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis 2019 Aug
- The $6 Million Drug Claim- New treatments for rare diseases are changing the lives of patients, but the price can reach millions of dollars for a single person.
K Thomas et al, NY Times, August 25, 2019
- Bleeding Disorders in Women: Free Materials
- The national blueprint for future basic and translational research to understand factor VIII immunogenicity: NHLBI State of the Science Workshop on factor VIII inhibitors.
Meeks Shannon L et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Jul 25(4) 595-602
- When innovation goes fast. The case of hemophilia.
Pilunni Daniela et al. Current opinion in pharmacology 2019 Jul
- The Community Counts Data Visualization Tool
- Molecular Genetics of von Willebrand Disease in Korean Patients: Novel Variants and Limited Diagnostic Utility of Multiplex Ligation-Dependent Probe Amplification Analyses.
Kim Hee Jung et al. Annals of laboratory medicine 2019 Nov 39(6) 545-551
- New Opportunities and Challenges in Hemophilia Care: 2019 Update
CDC Public Health Seminar, June 6, 2019
- Considering the benefits of newborn screening for haemophilia.
Moorehead Paul C et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 May
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Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
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- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
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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.