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.
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Results from two Phase 3 trials support the use of emicizumab for the preventive treatment of hemophilia A in children, adolescents, and adults, Genentech announced. These results will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting July 8-13 in Berlin, Germany. Emicizumab is an…
Grab the factor, syringes, butterfly needles, drapes, Band-Aids, self-adherent wrap, sharps container, and tourniquet! These things are usually what comes to mind when we think about hemophilia care. Medical supplies are the first thing we think of when we plan for day-to-day life with hemophilia. Preventing and treating bleeds…
Researchers have developed a biodegradable capsule to deliver a protein missing in patients with hemophilia B, according to a new study published in the International Journal of Pharmaceutics. If it proves to be successful, this capsule may provide a cheaper and less painful option to injections or infusions. The…
An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported. The report, titled…
More than 12,300 hemophilia cases in developing countries were treated with therapy donations from Bioverativ and its partner Sobi in 2016, Bioverativ said in a press release about its participation in World Hemophilia Day. The company, which is promoting genetic testing in women and girls who could be susceptible to bleeding episodes, worked with other…
Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or decrease the occurrence of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. The application is based on…
Novo Nordisk expects a decision this month by the U.S. Food and Drug Administration (FDA) on its product N9-GP  (nonacog beta pegol) as a treatment for hemophilia B. Approval of the drug may depend on the conclusions the FDA makes regarding safety concerns raised by its blood products advisory committee,…
Decades ago most hemophilia patients died young. Advances in treatment have led to them living about as long as the general population, which means that for the first time, doctors are facing the challenge of caring for aging hemophiliacs. A key problem is that in the days before treatment breakthroughs,…
It’s easy to spot physical damage, such as swollen joints, nosebleeds, limps, and bruises, when one suffers from a bleeding disorder. But the psychological and emotional damage that may result is not as easy to see or treat. A U.S. survey of hemophilia patients, published a few years…