Nine-year follow-up of gene therapy in haemophilia B confirms long-term safety and reduced bleeding, though full capsid ...

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 ...

Researchers complete final analysis of a phase 3 study of men with hemophilia B receiving intravenous etranacogene dezaparvovec.

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 ...

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 ...

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 FDA approved UniQure’s Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency).

Panel A shows the factor IX:C level after the infusion of BBM-H901, a factor IX–Padua gene product, but before the operation. The activity level of the dotted line is 40 IU per deciliter. The left ...

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 ...

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 ...

Idelvion (coagulation factor IX [recombinant], albumin fusion protein) is a prescription drug used to treat and help prevent bleeding episodes in people with hemophilia B. Idelvion is given as an ...