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 ...
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 ...
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 ...
KING OF PRUSSIA, Pa., Dec. 7, 2025 /PRNewswire/ -- Global biopharma leader CSL (ASX:CSL; USOTC:CSLLY) today announced five-year (60-month) results from the pivotal Phase 3 HOPE-B study, confirming the ...
A five-year study suggests gene therapy may help reduce bleeding and infusions as a hemophilia B treatment option.
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, ...
Intellia Therapeutics is opting out of a hemophilia B program, leaving the CRISPR/Cas9-based gene editing work on factor IX treatments in the hands of partner Regeneron. The bigger biotech had already ...
Hemophilia B, also known as Christmas disease, is a bleeding disorder. Its characteristic feature is a deficiency of clotting factor IX. This is a protein present in the blood that helps with ...
The indications, pharmacokinetics, and therapeutic guidelines for available coagulation products are reviewed. Patients with hemophilia, von Wille-brand's disease (VWD), or acquired inhibitors to ...