News

Swedish Orphan Biovitrum AB recently announced that Eloctate (efmoroctocog alfa; brand name Elocta in Europe), a treatment for hemophilia A, has been approved for reimbursement in the United Kingdom, Italy, and France. These countries join others in the European Union — Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands,…

The initial treatment in patients newly diagnosed with hemophilia B should definitely be done in hospital according to a study conducted by scientists in Turkey.

Researchers reviewed the use of high-purity concentrates containing two plasma molecules that are lacking in patients with Von Willebrand disease (VWD), a hemorrhagic disorder. These products may be a good strategy for managing pediatric patients with VWD and hemophilia A (HA) who do not respond to other treatments. The study, “Human Von…

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…

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.