The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important ...
Gene therapy for hemophilia B allowed almost three-fourths of patients to discontinue prophylactic factor IX therapy with no increase in bleeding, results of the pivotal BENEGENE-2 trial showed. The ...
Robert S. Gronke, William J. VanDusen, Victor M. Garsky, John W. Jacobs, Mohinder K. Sardana, Andrew M. Stern, Paul A. Friedman Proceedings of the National Academy of ...
Factor IX concentrate products ( Table 1) may be used to manage an acute hemorrhage or to decrease the risk of bleeding associated with surgery in patients with hemophilia B. Before factor IX ...
VON Willebrand's disease is manifested by a deficiency of factor VIII (antihemophilic factor) and a prolonged bleeding time and is inherited as an autosomal dominant trait of variable penetrance. 1 ...
A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results ...
Hemophilia B causes a deficiency in factor IX, a protein necessary for blood clotting. Replacement factor IX therapy is the gold standard treatment, but novel and genetic therapies are showing promise ...
Hemophilia B, also known as Christmas disease, is a bleeding disorder. Its characteristic feature is a deficiency of clotting factor IX. This is a protein present in the blood that helps with ...
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