Abstract |
Hemophilia B is a rare hereditary hemorrhagic disorder characterized by deficiency of the clotting factor IX (FIX). Hemophilia B patients experience mild-to-severe bleeding complications according to the degree of FIX defect. The mainstay of treatment of hemophilia B consists of replacement therapy and nowadays several plasma-derived and recombinant FIX products are commercially available. This article reviews the current management of hemophilia B patients analyzing the results of the most important clinical trials. In addition, it will focus on the more recent advances in the production of new FIX molecules aimed at the improvement of the clinical management of such patients.
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Authors | Massimo Franchini |
Journal | Expert review of hematology
(Expert Rev Hematol)
Vol. 7
Issue 5
Pg. 573-81
(Oct 2014)
ISSN: 1747-4094 [Electronic] England |
PMID | 25112898
(Publication Type: Journal Article, Review)
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Chemical References |
- Blood Coagulation Factor Inhibitors
- Recombinant Fusion Proteins
- Recombinant Proteins
- Polyethylene Glycols
- Factor IX
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Topics |
- Blood Coagulation Factor Inhibitors
(metabolism)
- Factor IX
(adverse effects, immunology, therapeutic use)
- Genetic Therapy
- Half-Life
- Hemophilia B
(drug therapy)
- Humans
- Polyethylene Glycols
(chemistry)
- RNA Virus Infections
(etiology)
- Recombinant Fusion Proteins
(pharmacokinetics, therapeutic use)
- Recombinant Proteins
(adverse effects, immunology, therapeutic use)
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