Designing ATHN 7: Safety, effectiveness and practice of treatment of people with haemophilia in the United States through a natural history cohort study

Haemophilia A and B are X-linked inherited bleeding disorders, resulting in the deficiency of clotting factor VIII and IX, respectively. Since the introduction of recombinant clotting factor concentrates in the early 1990s, the major safety concern for haemophilia therapy has been the development of...

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Bibliographic Details
Published in:Journal of Haemophilia Practice 2023-01, Vol.9 (1), p.147-155
Main Authors: Buckner, Tyler W., Daoud, Nabil, Miles, Moses, O'Neill, Carrie, Watson, Crystal, Recht, Michael
Format: Article
Language:English
Subjects:
effectiveness
haemophilia a
haemophilia b
natural history
safety
therapeutics
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Summary:Haemophilia A and B are X-linked inherited bleeding disorders, resulting in the deficiency of clotting factor VIII and IX, respectively. Since the introduction of recombinant clotting factor concentrates in the early 1990s, the major safety concern for haemophilia therapy has been the development of inhibitory antibodies, or inhibitors. Over the recent past, new therapies for the treatment and prevention of bleeding have received regulatory approval or are under study.
ISSN:2055-3390
2055-3390
DOI:10.2478/jhp-2022-0020