Hemophilia A
What's New
Last Posted: Jul 18, 2024
- Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A
- 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 - Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid.
Antal Zemplenyi et al. Pharmacoeconomics 2023 - Exploring factors impacting haplotype-based noninvasive prenatal diagnosis for single-gene recessive disorders.
Lingrong Kong et al. Clin Genet 2023 - Psychological Burden Among Patients With Inherited Bleeding Disorders in Madinah Province, Saudi Arabia.
Mohammed A Zolaly et al. Cureus 2023 15(9) e45165 - 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 8 (9) 853-856 - Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
Tadashi Matsushita et al. N Engl J Med 2023 8 (9) 783-794 - 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 - 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 - The FDA just approved another gene therapy. Here’s what to know about them
SA Yonah, Washington Post, July 1, 2023 - 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 - 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 - 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 4 (10386) 1427-1437 - 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 - Hemophilia A Gene Therapy - Some Answers, More Questions.
Lindsey A George et al. The New England journal of medicine 2023 2 (8) 761-763 - The ethics of gene therapy for hemophilia: a narrative review.
Lieke Baas et al. Journal of thrombosis and haemostasis : JTH 2023 - Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study.
Toshko Lissitchkov et al. Blood advances 2021 11 (4) 1089-1094 - Sources of Innovation in Gene Therapies - Approaches to Achieving Affordable Prices.
Kerstin N Vokinger et al. The New England journal of medicine 2023 1 - 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 - 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 - 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 - 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 - 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 - Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022 - Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options.
Regling Katherine et al. Pediatric health, medicine and therapeutics 2022 1327-35 - Disease of the Week: Hemophilia
CDC, March 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 - 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 - Precision Public Health in Action: New CDC Pilot Projects Integrating Human Genomics into Public Health Surveillance and Applied Research
M Clyne et al, CDC Blog Post, February 14, 2022 - 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 - The Spectrum of Pathogenic Variants in Iranian Families with Hemophilia A.
Azadmehr Sarah et al. Archives of Iranian medicine 2022 24(12) 887-896 - 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 - Gene therapy for hemophilia A
K O'Leary, Nature Medicine, November 30, 2021 - Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021 - 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 - Gene therapy for hemophilia: Current status and laboratory consequences.
Batty Paul et al. International journal of laboratory hematology 2021 43 Suppl 1117-123 - Prediction of hemophilia A severity using a small-input machine-learning framework.
Lopes Tiago J S et al. NPJ systems biology and applications 2021 7(1) 22 - Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.
Hart Daniel P et al. Orphanet journal of rare diseases 2021 16(1) 189 - 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 - Health care resource utilization and costs among adult patients with hemophilia A on factor VIII prophylaxis: an administrative claims analysis.
Croteau Stacy E et al. Journal of managed care & specialty pharmacy 2021 27(3) 316-326 - Evaluation of CDC's Hemophilia Surveillance Program - Universal Data Collection (1998-2011) and Community Counts (2011-2019), United States.
Schieve Laura A et al. Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002) 2020 Sep 69(5) 1-18 - BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
Konkle Barbara A et al. The New England journal of medicine 2020 Sep 383(11) 1018-1027 - Prevalence of FVIII Inhibitors Among Children with Hemophilia A: Experience at the Jordanian Royal Medical Services.
Oudat Raida et al. Medical archives (Sarajevo, Bosnia and Herzegovina) 2020 Jun 74(3) 187-190 - Beliefs and Values About Gene Therapy and In-Utero Gene Editing in Patients with Hemophilia and Their Relatives.
Vasquez-Loarte Tania C et al. The patient 2020 Aug - How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease.
Leebeek Frank W G et al. Blood 2020 Aug
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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
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 Public Health Genomics Branch 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:Feb 1, 2024
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