Catalyst Biosciences announced positive interim results from a Phase 2/3 clinical trial (NCT03407651) of the Factor VIIa variant marzeptacog alfa (activated) (MarzAA) in the preventative treatment of patients with hemophilia A or B with inhibitors. The study, aiming to include up to 12 patients, is recruiting in Russia…
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More than 21 years ago, when my first child was born and diagnosed with severe hemophilia A, he was very fortunate to have first-generation clotting factors available. These were concentrates stabilized with albumin that were very safe. When the factor was reconstituted with sterile water, a seemingly…
The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…
Under-the-skin, preventive treatment with Dalcinonacog alfa (DalcA) led to sustained and protective levels of factor IX (FIX) in people with severe hemophilia B, final data from a Phase 2b trial show. The findings, Phase 2b Trial to Evaluate the Safety and Factor IX Levels of a Daily Subcutaneous Prophylaxis…
The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.
A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…
A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their healthcare team in real time. MORE: Five tips for living healthily with hemophilia. The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing…
A novel clinical study called “The Irish Personalized Approach to the Treatment of Hemophilia (iPATH)” will seek to investigate the potential of new personalized treatment approaches in hemophilia by tailoring care based on the needs of individual patients. The collaborative study will be led by the Irish Hemophilia Society,…
The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A. Wilate, developed and marketed by Octapharma, is an injectable medication that contains human plasma-derived von Willebrand Factor (VWF)…
I recently attended a webinar called “Impact of Factor 8 on Bone Mineralization,” sponsored by Factor My Way, a patient support program. It featured Claudio Sandoval, MD, who has over 30 years of experience treating people with bleeding disorders and is passionate about bone health, an often overlooked aspect of…