The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…
Ixinity remains readily available to treat people with hemophilia B despite the current COVID-19 outbreak, Medexus Pharmaceuticals has announced. The company, which acquired Ixinity from Aptevo Therapeutics, also said it will continue working to ensure continued supply throughout the pandemic. “We would like to reassure the patients…
Poor joint health is associated with a higher risk of low self-esteem in adolescents with hemophilia, a study has found. The study, “Assessment of Self-Image With the Offer Self-Image Questionnaire in Adolescents With Hemophilia: A Single-Center Experience,” was published in the Journal of Pediatric Hematology/Oncology.
Prophylactic, or preventive, use of Hemlibra (emicizumab) promotes joint health in people with hemophilia A, according to a new analysis of data from a Phase 3 trial. These findings were announced in the oral presentation “Bone and Joint Health Markers in Persons with Hemophilia A (PwHA)…
A single infusion of AMT-061 (etranacogene dezaparvovec), uniQure’s experimental gene therapy for the treatment of hemophilia B, led to increased and sustained activity of clotting factor IX (FIX), according to one-year data from a Phase 2b clinical trial. The therapy effectively prevented…
Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…
The Analysis Group has partnered with China’s Institute of Hematology & Blood Diseases Hospital (IHBDH) to create the first blood disease research platform in that country. The new platform, called the National Longitudinal Cohort of Hematological Diseases in China (NICHE), will store clinical data on more than 10,000 people…
Direct‐acting antiviral (DAA) therapy is especially effective at eliminating hepatitis C virus (HCV) in people with bleeding disorders, including hemophilia, a study has found. The study, “Elimination of hepatitis C virus infection in patients with haemophilia in Belgium: A single‐centre experience,” was published in Haemophilia.
Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies have agreed to work together on a three-year research project that will focus on the identification and…
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