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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)
Last data update: Mar 28, 2024
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Hemophilia[original query]>>Original Studies[Product Type]
Towards Personalized Treatment in Hemophilia: The Role of Genetic Factors in Iron and Heme Control to Identify Patients at Risk for Haemophilic Arthropathy
LFD van Vulpen et al, JPM, January 26, 2024
KNOWLEDGE AND ATTITUDES TOWARDS GENE THERAPY OF A COHORT OF ITALIAN PATIENTS WITH HAEMOPHILIA.
Ilaria Cutica et al. J Thromb Haemost 2023
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Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid.
Antal Zemplenyi et al. Pharmacoeconomics 2023
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Exploring factors impacting haplotype-based noninvasive prenatal diagnosis for single-gene recessive disorders.
Lingrong Kong et al. Clin Genet 2023
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Psychological Burden Among Patients With Inherited Bleeding Disorders in Madinah Province, Saudi Arabia.
Mohammed A Zolaly et al. Cureus 2023 15(9) e45165
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Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
Tadashi Matsushita et al. N Engl J Med 2023 389(9) 783-794
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Association of Genetic Variant FVIII Gene and Factor VIII: A Pilot Study Among Hemophilia A Female Relatives in Saudi Arabia.
Abdullah T Almohammadi et al. Cureus 2023 15(7) e42038
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Development of a haemophilia A gene therapy shared decision-making tool for clinicians.
Jacqueline Limjoco et al. Haemophilia 2023
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Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.
Jacqueline Limjoco et al. Patient Prefer Adherence 2023 171093-1105
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Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial.
Guy Young et al. Lancet 2023 401(10386) 1427-1437
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Trends in prescribing practices for management of haemophilia: 1999-2021.
Randall Curtis et al. Haemophilia : the official journal of the World Federation of Hemophilia 2023
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Expectation and loss when gene therapy for haemophilia is not an option: An exigency sub-study.
Simon Fletcher et al. Haemophilia : the official journal of the World Federation of Hemophilia 2023
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The emotional experience of mothers of children with haemophilia: maternal guilt, effective coping strategies and resilience within the haemophilia community.
Nina Sheridan et al. Haemophilia : the official journal of the World Federation of Hemophilia 2023
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Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study.
Toshko Lissitchkov et al. Blood advances 2021 6(4) 1089-1094
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Gene therapy preferences and informed decision-making: Results from a National Hemophilia Foundation Community Voices in research survey.
Limjoco Jacqueline et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.
Castaman Giancarlo et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Challenges and knowledge gaps facing hemophilia carriers today: Perspectives from patients and health care providers.
Chaigneau Megan et al. Research and practice in thrombosis and haemostasis 2022 6(6) e12783
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Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy.
Quinn Jennifer et al. Patient related outcome measures 2022 13169-180
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Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future Hemophilia Genotyping Initiative in the United States.
Johnsen Jill M et al. Journal of thrombosis and haemostasis : JTH 2022
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Examining patient and professional perspectives in the UK for gene therapy in haemophilia.
Woollacott Ione et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022
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Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
S Fong et al, Nature Medicine, April 11, 2022
Changing paradigms of hemophilia care across larger specialized treatment centers in the European region.
Windyga Jerzy et al. Therapeutic advances in hematology 2022 1320406207221088462
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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022
Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with hemophilia A.
Klamroth Robert et al. Human gene therapy 2022
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Parvovirus 4 in Individuals with Severe Hemophilia A and Matched Control Group.
Asiyabi Sanaz et al. International journal of hematology-oncology and stem cell research 2022 15(3) 192-198
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The Spectrum of Pathogenic Variants in Iranian Families with Hemophilia A.
Azadmehr Sarah et al. Archives of Iranian medicine 2022 24(12) 887-896
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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021
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
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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
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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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Page last reviewed:
Feb 1, 2024
Page last updated:
Mar 28, 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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