Researchers complete final analysis of a phase 3 study of men with hemophilia B receiving intravenous etranacogene dezaparvovec.
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 ...
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 ...
Forbes contributors publish independent expert analyses and insights. Joshua Cohen is a Boston-based writer who covers health policy. On November 22 nd, the Food and Drug Administration (FDA) approved ...
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 ...
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 ...
SOUTH SAN FRANCISCO, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (Nasdaq:CBIO), a clinical-stage biopharmaceutical company focused on developing novel medicines to address ...
“ Despite several major advances in treatment options for people with hemophilia B, including extended-release recombinant factor IX biologics and the availability of a new gene therapy, significant ...
KING OF PRUSSIA, Pa., Dec. 10, 2022 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) today announced the presentation of data affirming the long-term durability and safety of single-infusion ...
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, ...