Hemlibra (emicizumab) was able to prevent excessive bleeding in patients with severe hemophilia A without inhibitors, according to results from a Phase 3 trial. Trial findings were published in the study, “Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors,” in The New England Journal of…

The U.S. Food and Drug Administration has approved Bayer’s Jivi, previously known as BAY94-9027, as a preventive therapy for hemophilia A in patients ages 12 and older, the company announced. The recommended regimen is for Jivi to be administered intravenously (into the blood) two times a week (30-40 IU/kg),…

I’m looking at you, insurance companies and banks who won’t approve people who have a history of chronic illness or who were born with one — such as my husband, who has hemophilia. Of course, I am looking at this from a different perspective than the one businesses take. Then…

uniQure has treated the first patient in a Phase 2b dose-confirmation study of its investigational gene therapy, AMT-061, for people with severe and moderately severe hemophilia B. The trial is still recruiting participants. AMT-061 uses a viral vector, AAV5, to deliver the gene for a mutated clotting factor…

I’ve always had a hard time coping with the fact that it’s quite difficult for me to find a decent source of income due to hemophilia and epilepsy. Although government and private institutions say that they are inclusive of persons with disabilities (PwDs), the reality is that discriminations against…

Caeleb is super excited about starting the seventh grade this week. He loves the new town we are living in and is hopeful for the school year. He wants to play basketball. Now, most people would say that is a normal thing for a 12-year-old boy to want…

The Royal College of Surgeons in Ireland (RCSI) and Bayer will collaborate to develop improved treatments for patients with severe hemophilia. The research collaboration will focus on personalized treatments adapted to the severity of each patient’s condition to achieve safer and more effective blood clotting in hemophilia…

Catalyst Biosciences announced positive interim results from a Phase 2/3 clinical trial (NCT03407651) of the Factor VIIa variant marzeptacog alfa (activated) (MarzAA) in the preventative treatment of patients with hemophilia A or B with inhibitors. The study, aiming to include up to 12 patients, is recruiting in Russia…

Health Canada has approved Hemlibra (emicizumab) as a treatment to prevent or reduce bleeding in Hemophilia A patients in Canada who have factor VIII inhibitors. Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the…

  My eldest son was about to celebrate his ninth birthday. He had attended a fantastic performing arts camp. My wife and I bought Happy Meals for the entire ensemble in honor of “MacDonald the Elder’s” special day. We had an announcement that we couldn’t wait to share…