Hemophilia is a blood disorder that is characterized by a shortage of specific clotting factors that help to form blood clots and stop bleeding. The primary treatment for hemophilia is replacement therapy that supplies the clotting factors to hemophilia patients who lack them. Other approved hemophilia treatments include…
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Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…
Hemlibra (emicizumab) is an effective and less costly therapy for hemophilia A patients who have substances in their blood that prevent clotting factor therapies from working as well as they should, according to a therapy rating organization. The Institute for Clinical and Economic Review (ICER) updated an initial report on Hemlibra…
Doctors in Canada are proposing a set of measures to better judge outcomes and the success of programs helping teen-age hemophilia patients transition to adult care, especially for those with more severe disease. They also listed 15 “key elements” of a successful pediatric-to-adult transition program, including eight the doctors saw as feasible…
I often tell people that my mind is a dangerous neighborhood and that I should never venture into it alone. This usually gets a laugh, which the comic in me appreciates, as I often say it to lighten a difficult conversation. A little release of tension doesn’t hurt anyone. But…
The first participant has been dosed in a Phase 1/2 dose-confirmation trial that is testing FLT180a, an experimental gene therapy that Freeline is developing to treat hemophilia B. “We are very excited about this progress in our hemophilia B program as our enhanced focus on execution is starting to…
I have seen it many times. When a woman in a family with hemophilia gets pregnant, some feel OK emotionally, but for many, it causes a moment of panic, and sometimes sheer terror. What have they done? They are so scared that they passed…
Let me tell you one of my secrets: I have anxiety. I don’t mean the kind of anxiety you’d feel if a package hadn’t arrived days past a delivery date. Or if you were lost in an unfamiliar place at night trying to find your way home. That kind of…
As a woman affected by the bleeding disorders hemophilia B and von Willebrand disease, I realize that increasing awareness about bleeding disorders in undiagnosed women and girls is essential. All women should be aware of the symptoms of a bleeding disorder and be tested if warranted. Bleeding disorders, even mild…
There’s something special about each day of the week. I enjoy Thursday because my weekend begins at the close of the day. I enjoy Wednesdays because it’s the middle of the week, and the weekend is closer (I think there’s a pattern here). Sundays are special because, as a…