Hemophilia
What's New
Last Posted: Nov 18, 2021
- Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021 - Real Stories from People Living with Hemophilia – Carrie
CDC, November 2021 - Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations.
Garrison Louis P et al. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research 2021 24(11) 1628-1633 - Why is AAV FVIII gene therapy not approved by the US Food and Drug Administration yet?
Arruda Valder R et al. Blood advances 2021 5(20) 4313 - Emerging drugs for hemophilia A: insights into phase II and III clinical trials.
Kizilocak Hande et al. Expert opinion on emerging drugs 2021 - What is a ‘serious’ genetic condition? The perceptions of people living with genetic conditions
FK Boardman et al, EJHG, September 27, 2021 - [Current developments in hemostatic treatment for patients with hemophilia].
Nogami Keiji et al. [Rinsho ketsueki] The Japanese journal of clinical hematology 2021 62(8) 1213-1221 - A qualitative study on the experiences of haemophilia carriers before, during and after pregnancy.
Punt Marieke C et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021 - Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Hemophilia B Without Inhibitors in China.
Liu Guoqing et al. Clinical therapeutics 2021 - Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A.
Pasi K John et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
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About Rare Diseases PHGKB
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 (16)
- CDC Publications (12)
- Human Genome Epidemiologic Studies (154)
- Genomic Tests Evidence Synthesis (7)
- Genomic Tests Guidelines (5)
- Tier-Classified Guidelines (4)
- Pathogen Advanced Molecular Detection (2)
- State Public Health Genomics Programs (7)
- Reviews/Commentaries (76)
- Tools/Methods (3)
- Ethical/Legal and Social Issues (ELSI) (4)
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, 2021
- Page last updated:Nov 28, 2021
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