Hemophilia, a disorder characterized by excessive bleeding, is caused by the lack of activity of certain clotting factors, which are proteins that are needed to form blood clots.
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SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
BAY 94‐9027, an investigational factor VIII replacement therapy being developed by Bayer for severe hemophilia A, is able to stay longer in patients’ blood than other standard therapies, according to a study analyzing data from three…
Long-term treatment with Esperoct (turoctocog alfa pegol) prevented bleeding in one-fifth of children with severe hemophilia A over the course of a Phase 3 clinical trial, according to final results from the study. Treatment was effective in both spontaneous and traumatic bleeds, and appeared to be…
Bleeding episodes in people with severe hemophilia A receiving prophylactic (preventive) treatment have the potential to impair patients’ health-related quality of life, according to data from a survey conducted in Europe. Even a single bleed was linked to marked medication-related and non-related health costs. In fact, in terms of…
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How to Explain Hemophilia to Children
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
Social Clips
How to Explain Hemophilia to Children
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
The U.S. Food and Drug Administration (FDA) has granted priority review to the therapy emicizumab for patients with hemophilia A with factor VIII inhibitors, Genetech, the drug’s developer, announced. The FDA also announced the acceptance of Genetech’s Biologics License Application (BLA) for emicizumab prophylaxis (preventive) therapy in adults, adolescents and children…
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CSL Behring’s Afstyla Allows Hemophilia A Patients to Use Less Factor Replacement, Analysis Shows
Hemophilia A patients treated with CSL Behring’s Afstyla (recombinant factor 8 single-chain) require less factor to control bleeding compared to those taking octocog alfa, the company recently announced. The lower consumption holds both for patients using clotting factors on-demand and those who require preventive, or prophylactic treatment. CSL Behring…
SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…