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…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

When my youngest son, Caeleb, was 8 years old, our family went on a trip that required him to use a wheelchair at the airport. His severe hemophilia A with an inhibitor had caused persistent joint bleeds, which resulted in significant pain and limited mobility, as well as…

Not long after I started working at the Hemophilia Federation of America (HFA), the organization hosted a congressional reception to help raise awareness about bleeding disorders. The bleeding disorders community must maintain its visibility among government decision-makers who help address important issues, such as ensuring our access to the…

The U.S. Food and Drug Administration (FDA) has approved an update to the label of Alprolix, based on long-term efficacy and safety data from two Phase 3 studies, the B-YOND extension trial and the Kids B-LONG pediatric study, manufacturer Bioverativ announced. This label extension, which adds pediatric data, further supports…