I’m looking forward to attending the Hemophilia Federation of America’s annual symposium, running today through Saturday. I’ll be among the community I consider family. But it’s been a few years since I attended a large conference. I’m excited about reconnecting with old friends, but I’m also a bit…

The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…

Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using concizumab as a prophylactic (preventive) treatment compared with those who weren’t on any prophylaxis. That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which…

In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…

My 13-year-old son Caeleb, who had severe hemophilia A and an inhibitor, has not had a bleed in five years. For three of those years, he received a daily infusion with a plasma-derived product, and for the past two years, he has been on a treatment that…

I’m starting to recover from my recent iliopsoas bleed. I’m irritated that my right hip isn’t as flexible as it used to be, but that’s a normal part of recovery. It’ll take me a while to regain the ability to move freely and easily. I’m looking forward to the…

Eloctate can induce immune tolerance in people with hemophilia A more quickly than other regimens, a study suggests. The study, “Real‐world data of immune tolerance induction using recombinant factor VIII Fc fusion protein in patients with severe haemophilia A with inhibitors at high risk for…

Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…