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 ...
At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...
A long-acting coagulation factor treatment of patients with hemophilia B is safe and effective for preventing bleeding events, according to an article published online December 4 and in the December ...
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 ...
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 ...
Mean factor IX activity level remained stable at 36.1 ± 15.7 IU/dL at year 5, with 80% of participants maintaining levels ≥ 12 IU/dL. Adjusted annualized bleeding rates decreased from 4.16 during lead ...
Afghanistan is facing a critical shortage of life-saving blood clotting factors, leaving hundreds of haemophilia patients at the risk of uncontrolled internal bleeding, permanent disability, and ...
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 ...
After blood vessel damage, effective blood clotting is essential to halt bleeding. However, this process is inefficient in some individuals due to hereditary factors. Hemophilia B, for example, ...
After blood vessel damage, effective blood clotting is essential to halt bleeding. However, this process is inefficient in some individuals due to hereditary factors. Hemophilia B, for example, ...
There are several lab tests for hemophilia. However, the main tests detect the clotting activity of factors VIII and IX, which are proteins in the blood. Hemophilia is a bleeding disorder in which an ...
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