As gene therapy is relatively new treatment option for hemophilia B, it's normal to have some questions about it. Here are answers to some commonly asked questions about gene therapy for hemophilia B.
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The U.S. Food and Drug Administration (FDA) has given fast track designation to ASC618, an investigational one-time gene therapy for hemophilia A in the pipeline of ASC Therapeutics. Fast track status works to speed the development and regulatory review of treatments for serious disorders that show a potential to fill…
Nuwiq (simoctocog alfa) is an approved therapy that works to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
The prevalence of hypertension is higher in patients with hemophilia than in the general population, according to a nationwide study in Sweden with long-term follow-up. In contrast, no differences were seen in the prevalence of angina pectoris and myocardial ischemia, two cardiovascular diseases. The study, “…
When given at low doses, extended half-life factor IX concentrates Alprolix (eftrenonacog alfa) and Idelvion (albutrepenonacog alfa) can prevent spontaneous bleeds in patients with severe hemophilia B, a study has found. The study, “A single centre retrospective study of low dose prophylaxis with…
Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…
When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…
Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for hemophilia A and B. Hemophilia is caused by a lack of blood clotting factors or their faulty production,…
Hemophilia can affect many aspects of life, including sexual health. If you have hemophilia, sexual activity is generally safe as long as certain precautions are taken.
The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…