Mononine, or human coagulation factor IX, is a factor IX concentrate indicated for the prevention and control of bleeding in hemophilia B. The U.S. Food and Drug Administration (FDA) approved its use in 1992, making it the first highly purified factor IX on the U.S. market.
Spark Therapeutics will present novel results regarding the ongoing Phase 1/2 clinical trial (NCT02484092) investigating SPK-9001 in hemophilia B patients at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, to be held July 8-13 in Berlin. Two presentations focus on data from the trial: “SPK-9001:…
Cellphones are a good way for hemophiliacs receiving care at home to relay information that doctors need to stay on top of their treatment. But a lot of patients have yet to embrace the service — and a recent online survey of 181 hemophiliacs shed some light on why. Spanish…
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…
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.
Patient treatment for hemophilia B has come a long way since the introduction of factor IX (FIX) concentrates. In a recent commentary published in the journal Blood and titled “A new era for hemophilia B treatment,” researchers from Oregon Health and Science University and Bloodworks Northwest highlighted recent advances in…
Ruptured spleens are rare in newborns, usually occurring secondary to an underlying coagulation disorder, such as hemophilia, or a splenic abnormality. Here, researchers report the first case of a newborn with a spontaneous splenic rupture and severe hemophilia A who was successfully treated without surgical intervention. The case study, “…
The European Medicines Agency (EMA) is considering lowering the minimum number of patients required in clinical trials of potential hemophilia treatments because of “dramatic” increases in hemophilia research and concerns about the availability of patients for rare diseases like hemophilia A. “EMA began the process after becoming concerned that the minimum…
Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016Â World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…
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