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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Precision Health Database|Search|Public Health Genomics and Precision Health Knowledge Base (PHGKB)
Heart, Lung, Blood and Sleep Disorders
Last data update: Apr 25, 2024
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Hemophilia[original query]>>Evidence Synthesis [Product Type]
The effectiveness and value of gene therapy for hemophilia: A Summary from the Institute for Clinical and Economic Review's California Technology Assessment Forum.
Jeffrey A Tice et al. J Manag Care Spec Pharm 2023 29(5) 576-581
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Global epidemiology of factor XI deficiency: A targeted review of the literature and foundation reports.
Zhang Xinruo et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A.
Ten Ham Renske M T et al. HemaSphere 2022 6(2) e679
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CLINGEN Actionability Report for von Willebrand Disease - VWF
ClinGen Actionability Working Group
CLINGEN Actionability Report for Factor XI Deficiency - F11
ClinGen Actionability Working Group
CLINGEN Actionability Report for F5: Factor V deficiency (AR) - F5
ClinGen Actionability Working Group
CLINGEN Actionability Report for Hemophilia A and B - F8, F9
ClinGen Actionability Working Group
Gene therapy in hemophilia A: a cost-effectiveness analysis.
Machin Nicoletta et al. Blood advances 2018 Jul 2(14) 1792-1798
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Living with a rare disorder: a systematic review of the qualitative literature.
von der Lippe Charlotte et al. Molecular genetics & genomic medicine 2017 Nov 5(6) 758-773
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Cost-effectiveness analysis of carrier and prenatal genetic testing for X-linked hemophilia.
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Page last reviewed:
Feb 1, 2024
Page last updated:
Apr 25, 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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