News

Chinese researchers identified a new and simple method to identify women who may be carriers of the gene that causes hemophilia A.

Researchers from San Joaquin General Hospital in California reported the case of a 66 year-old man who was successfully treated for acquired hemophilia A and prostate cancer simultaneously.

In an article titled “Recognizing the need for personalization of hemophilia patient-reported outcomes in the prophylaxis era” published in the scientific journal Haemophilia, researchers reviewed two new tools that are used to measure patient reported outcome measures.

New research suggests that the main developmental role of heterochromatin, a form of dense DNA found in chromosomes, is to suppress virus-like DNA elements known as “jumping genes” from replicating and attaching themselves across a person’s genome, potentially destroying important genes and causing a variety of diseases, including hemophilia and cancer. The study, by University of North Carolina…

Sangamo BioSciences announced that its zinc finger nuclease (ZFN)-mediated genome editing product, SB-FIX, has received orphan drug designation from the U.S. Food and Drug Administration (FDA), speeding its development as a potential treatment for hemophilia B. Sangamo plans to initiate a Phase 1/2 clinical trial, SB-FIX-1501, in adults with this disease later…

The World Federation of Hemophilia (WFH) has created a universal case report form for the collection of core data on hemophilic patients in a standardized format that will include relevant diagnosis, therapies, and outcome. Previous WFH forms provided only an overview of the number of patients and access to treatment.

A number of clinical studies have reported that bone marrow-derived cells contribute to the production of clotting factor VIII (FVIII), the molecule linked to hemophilia A. Because the current treatment for this bleeding disorder is associated with some disadvantages and risk, hematopoietic stem cell transplant (HSCT) has become an emerging therapy approach…

Researchers in India recently reported the case of child with hemophilia and traumatic intracerebellar hemorrhage, who was successfully treated with transfusions of plasma. They recommend a conservative approach as a first line of treatment, before more invasive alternatives such as surgery. The case report, “Intracerebellar haemorrage in a haemophilia child,” was published in…

In hemophilia A, the most significant therapeutic complication is the development of antibodies against factor VIII (FVIII) that inhibit its coagulant activity and make the therapy ineffective. The plasma-derived porcine FVIII product represents a therapy with low levels of autoantibodies and successful management of bleeding when compared to human plasma-derived products.

CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…