Fresh Look at Mechanism Finds ‘Important Role’ in Factor VIII Regulation
A mechanism largely left unexplored was shown to help regulate factor VIII (FVIII) — the clotting protein that is missing or defective in people ... Read more
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‘In the Twinkling of an Eye’: The Moments That Change Our Lives
I started writing for BioNews, the publisher of Hemophilia News Today, almost three years ago. However, it recently dawned on me that I never ... Read more -
Roctavian, Hem A Gene Therapy, Sustains Drop in Bleeding Rates, Phase 3 Data Show
Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one ... Read more -
What Does Hemophilia Look Like?
What does an illness look like? Sometimes a bald head, pale skin, or a mobility device are outward signs that someone has an illness. ... Read more -
For Too Long, I Thought My Bleeding Was Normal
Two weeks ago, my daughter helped me move a small refrigerator. I had the front end and she had the back, which was open ... Read more
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NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life ... -
Parents, Lacking Information, Wary of Gene Therapies, UK Survey Finds
Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but ... -
Hemlibra Lowered Number of Hemophilia A Bleeds Over Nearly 6 Years
When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and ... -
Create an Action Plan for Your New Year’s Resolutions
In our culture, we learn at a very young age to make resolutions at ... -
Starting the New Year With a Focus on Helping My Son
A new year is always filled with possibilities and hope. After living through 2020, ...
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Europe’s CHMP Recommends Approval of Hemlibra to Treat Hemophilia A
News
Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds ... Read more