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 ...

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 ...

Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained ...

Hemophilia B, also called factor IX (FIX) deficiency, is an inherited condition that prevents blood from clotting normally. This means that if you experience a cut, scrape, or injury, the bleeding ...

Prophylactic treatment for hemophilia B necessitates lifelong, regular intravenous factor IX infusions. Gene therapy offers the possibility of a single-dose treatment that produces durable endogenous ...

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 ...

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 ...

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, ...

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 ...

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 ...