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Understanding types and therapies for rare blood disorders
Hemophilia: Hemophilia is a genetic disorder ... genetic disorder characterized by abnormal hemoglobin, causing red blood cells to become rigid and sickle-shaped, leading to blockages in blood ...
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After 4 years, hemophilia B bleeding rates remain low with Hemgenix
Four years after receiving Hemgenix in a Phase 3 trial, bleeding rates and the use of replacement therapy are low for men ...
The New England Journal of Medicine1mon
Platelet-Targeted Gene Therapy for Hemophilia A with Inhibitor History
Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia ... from the National Heart, Lung, and Blood Institute and a grant (UO1TR0018414) from the National Center ...