Results 301 to 310 of about 13,086,034 (342)
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Closing In on Treatment for Hemophilia B
New England Journal of Medicine, 2017Hemophilia is an X-linked recessive genetic bleeding disorder that occurs in two major forms. Approximately 80% of patients with hemophilia have mutations in the factor VIII clotting-factor gene (h...
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Hemophilia B Mutational Analysis
2003Since the cloning of the factor IX gene in 1982 (1), there have been several strategies employed for the identification of mutations in the mutationally heterogeneous hemophilia B population. Initially, such strategies inevitably employed Southern blotting to screen for gross deletions (2) or restriction site alterations (3), and cloning of the ...
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Human Gene Therapy Clinical Development, 2018
Gene therapy for severe hemophilia is on the cusp of entering clinical practice. However, there is limited clinical experience in this area given that gene transfer is a relatively recent technology.
Wolfgang A. Miesbach, E. Sawyer
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Gene therapy for severe hemophilia is on the cusp of entering clinical practice. However, there is limited clinical experience in this area given that gene transfer is a relatively recent technology.
Wolfgang A. Miesbach, E. Sawyer
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Molecular Analysis of Hemophilia B: »Greifswald Registry FIX Deficiency (Hemophilia B)«
2003Hemophilia B (HB) is due to multiple defects in the factor IX gene. More than 90% of mutants are single substitutions, small additions (
K. Wulff, F. H. Herrmann, W. Schröder
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Segregation analysis of hemophilia A and B.
American journal of human genetics, 1985We analyzed a sample of 1,485 families with hemophilia A and B and with unknown diagnosis. The frequency of sporadic cases was estimated to be .166 and .078 for the two types of hemophilia, respectively. The sex ratio of mutation rates did not differ significantly from unity. The average age of maternal grandfathers of probands at birth of mothers with
BARRAI, Italo Enrico +4 more
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Hemophilia B (Factor IX Deficiency).
Hematology/Oncology Clinics of North America, 2021R. Sidonio, L. Malec
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Hemophilia B Leyden and a Similar Variant of Hemophilia A
New England Journal of Medicine, 1982Inga Marie Nilsson, Rolf Ljung
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Marstacimab (Hympavzi) for Hemophilia A and B
The Medical Letter on Drugs and TherapeuticsMarstacimab-hncq (Hympavzi – Pfizer), a tissue factor pathway inhibitor (TFPI) antagonist, has been approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ≥12 years old who have hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors.
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