News

Researchers at the Mayo Clinic Arizona have reported a third rare case of hemophilic pseudotumor (HP) identified in a non-hemophiliac patient. Findings from the investigation, “Hemophilic pseudotumor in a non-hemophilic patient treated with a hybrid procedure of preoperative embolization of the feeding arteries followed by surgical resection—A…

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…