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 ...
Hemophilia A and B involve deficiencies in coagulation factors VIII and IX, with traditional treatments requiring frequent infusions. Nonfactor therapies, including Hemlibra and rebalancing agents, ...
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 ...
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 ...
Different types of assays led to different results in measures of factor IX variant FIX-R338L, the authors found. A new field study could help clinicians better interpret laboratory measurements of ...
Please provide your email address to receive an email when new articles are posted on . The novel gene therapy BAX 335 did not induce sustained factor IX expression among patients with hemophilia B, ...
The risk of an immune response to the coagulation factor IX (F.IX) transgene product is a concern in gene therapy for the X-linked bleeding disorder hemophilia B. In order to investigate the mechanism ...
Hemophilia B is a severe X-linked bleeding diathesis caused by the absence of functional blood coagulation factor IX, and is an excellent candidate for treatment of a genetic disease by gene therapy.
The projected cost reductions held both for patients switching from a different extended-half-life product and for patients switching from a standard-half-life product. Switching patients with ...
The US Food and Drug Administration (FDA) yesterday approved the first recombinant coagulation factor IX (Rixubis, Baxter Healthcare) for the routine prevention of bleeding in patients with hemophilia ...
If your child has hemophilia, their blood doesn't clot the way it should. If they have a cut, scrape, or other injury, they will bleed longer than other people do. The bleeding can happen on the ...
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