Last Posted: Mar 23, 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
- 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
- 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
- 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 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
- 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
- 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
- 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
- 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
- Current challenges in hemophilia genetics and how they can be overcome
JM Johnson, Nat Cardiovasc Research, October 2022
- 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
- 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
- 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
- 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
- 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
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
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- 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:Feb 1, 2023
- Page last updated:Mar 28, 2023
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