Fitusiran Dosing in Trials Reduced to Lower Blood-clot Risk
Sanofi has reduced the dosing of its investigational treatment fitusiran in clinical trials to lower the risk of blood clots in people with hemophilia, according ... Read more
Updates
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I Made the Mistake of Invalidating My Husband
It just slipped out that one time. I wasn’t even thinking. My husband, Jared, and I had been talking casually about how some of his ... Read more -
FDA Partially Rescinds Indications for BeneFIX and Ixinity
Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve BeneFIX and Ixinity for additional indications ... Read more -
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more -
Honoring Those Who Went Before Us
At every annual event sponsored by the National Hemophilia Foundation, a particular room is dedicated to remembering those who died from complications of hemophilia. In ... Read more
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Low Risk of Developing Inhibitors Found for Nuwiq Patients
Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies ... -
Why I Don’t Hate My Dad for Giving Me Hemophilia
My mom asked me last week if I ever curse my dad for giving me ... -
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ... -
Learning About Rare Diseases Broadened My Perspective on Health and Wellness
My husband, Jared, and I were recently talking about Rare Disease Day, which falls on ... -
Posture Exercises May Improve Joint Health in Children
Exercises to control posture could improve joint health in children with hemophilia and should form ...
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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 to ... Read more