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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Last data update: Apr 17, 2024
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Hemophilia[original query]>>Reviews/Commentaries[Product Type]
Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B.
Giancarlo Castaman et al. Expert Rev Hematol 2023
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How do people inherit hemophilia?
Medical News Today, September 2023
Hemostasis - A Balancing Act.
H Marijke van den Berg et al. N Engl J Med 2023 389(9) 853-856
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Researchers uncover mysteries behind immune response to hemophilia A treatment
Medical XPress, August 2023
Community Counts: Understanding Hemophilia and Other Bleeding Disorders through Public Health Surveillance
CDC webinar, August 24, 2023
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.
Duc Q Tran et al. Expert review of hematology 2023 16(sup1) 19-37
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Hemophilia A Gene Therapy - Some Answers, More Questions.
Lindsey A George et al. The New England journal of medicine 2023 388(8) 761-763
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The ethics of gene therapy for hemophilia: a narrative review.
Lieke Baas et al. Journal of thrombosis and haemostasis : JTH 2023
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Current challenges in hemophilia genetics and how they can be overcome
JM Johnson, Nat Cardiovasc Research, October 2022
Progress, and prospects in the therapeutic armamentarium of persons with congenital hemophilia. Defining the place for liver-directed gene therapy.
Di Minno Giovanni et al. Blood reviews 2022 101011
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Haemophilia gene therapy-Update on new country initiatives.
Reiss Ulrike M et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 461-67
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Is the world ready for gene therapy?
Garrison Louis P et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 25-8
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Precision Public Health in Action: Enhancing models to predict risk of adverse treatment outcomes in people with hemophilia
CJ Bean et al, CDC Blog Post, March 29, 2022
Prepare the Way for Hemophilia A Gene Therapy
CD Thornburg, NEJM, March 17, 2022
Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options.
Regling Katherine et al. Pediatric health, medicine and therapeutics 2022 1327-35
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Disease of the Week: Hemophilia
CDC, March 2022
Electronic diaries in the management of haemophilia gene therapy: Perspective of an expert group from the German, Austrian and Swiss Society on Thrombosis and Haemostasis (GTH).
Miesbach Wolfgang et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Health technology assessment for gene therapies in haemophilia.
O'Hara Jamie et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Hemophilia gene therapy: ushering in a new treatment paradigm?
George Lindsey A et al. Hematology. American Society of Hematology. Education Program 2021 2021(1) 226-233
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The Clinical Genetics of Hemophilia B (Factor IX Deficiency).
Miller Connie H et al. The application of clinical genetics 2021 14445-454
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Gene therapy for hemophilia A
K O'Leary, Nature Medicine, November 30, 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
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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
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Emerging drugs for hemophilia A: insights into phase II and III clinical trials.
Kizilocak Hande et al. Expert opinion on emerging drugs 2021
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[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
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Gene therapy for hemophilia: Current status and laboratory consequences.
Batty Paul et al. International journal of laboratory hematology 2021 43 Suppl 1117-123
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Hemophilia
E Berntorp et al, Nature Dis Primers., June 24, 2021
Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
Garrison Louis P et al. Journal of managed care & specialty pharmacy 2021 27(5) 674-681
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Data from CDC’s hemophilia surveillance programs can help prevent or reduce hemophilia-related health problems
CDC, March 2021
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Page last reviewed:
Feb 1, 2024
Page last updated:
Apr 17, 2024
Content source:
Public Health Genomics Branch in the Division of Blood Disorders and Public Health Genomics
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National Center on Birth Defects and Developmental Disabilities
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