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Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…

Prophylactic hemophilia treatment can save patients with severe forms of the disease from the long-term consequences of joint damage and other complications. A report by Dr. Suchitra S. Acharya, MD, of Hofstra Northwell School of Medicine in New York, gives a detailed overview of the current state and future perspectives…

Two presentations at the 2016 American Society of Hematology Annual Meeting (ASH) highlight ongoing treatment advances for hemophilia B. The data suggest that life with hemophilia could soon become easier to manage. Long-acting replacement therapy The ASH meeting, held in San Diego on Dec. 3-6, was a platform for CSL Behring to…

Researchers have created a model for how factor VIII replacement concentrates behave in the body of hemophilia A patients undergoing surgery, which could allow doctors to calculate more accurate and individualized doses for factor replacement treatment. The study “A Population Pharmacokinetic Model For Perioperative Dosing Of Factor VIII In Hemophilia A…

Key findings from clinical trials of three recombinant coagulation factors as new hemophilia treatments, presented at last year’s Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015), have been published in a supplemental issue of the journal Thrombosis Research. The results were presented in a session…

A hemophilia gene therapy developed by Spark Therapeutics and Pfizer nearly eliminated bleeding episodes in 10 patients who took part in a Phase 1/2 trial. The treatment, SPK-9001, worked so well in eight of the 10 that they were able to stop taking clotting factor replacements. Researchers published the preliminary trial…

Most young people with moderate or severe hemophilia A and B in France receive long-term prophylactic (preventive) treatment — an approach linked to a lower rate of bleeding episodes than among patients treated on demand. The study, “Choice of factor VIII/IX regimen in adolescents and young adults with…

Although gene therapy for hemophilia has reached clinical testing in humans, researchers continue to work toward improved delivery systems of the treatment — not an easy task considering the large size of the human factor VIII gene. In a study titled “Characterization of Adeno-Associated Viral Vector-Mediated Human Factor…

Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.

Tremeau Pharmaceuticals has received guidance from the U.S. Food and Drug Administration (FDA) on how to conduct a pivotal Phase 3 trial exploring rofecoxib (TRM-201) in patients with hemophilia-associated joint pain. The advice was offered during a formal meeting with the agency, where both parties agreed on the design…