Sigilon Therapeutics’s Afibromer SIG-003 candidate for the long-term treatment of patients with hemophilia B recently received an Advanced Therapy Medicinal Product (ATMP) designation in the European Union. The designation was granted by the Committee for Advanced Therapies of…
The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…
Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers at the Children’s Hospital of Philadelphia offered a rundown on the advances in an article in the journal Blood titled “Novel approaches to hemophilia therapy: successes and challenges.” They include …
Bioverativ will collaborate with the Boston imaging company Invicro to use ultrasound imaging to improve the diagnosis and management of joint disease in hemophilia patients. “Through this innovative collaboration with Invicro, we hope to create meaningful progress in hemophilia care by helping people with hemophilia, their caregivers, physicians and treatment team…
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…
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