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…

A mechanism largely left unexplored was shown to help regulate factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a study in mice. While early and preclinical work, it is reported to be the first study of this mechanism…

Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one year, top-line data from a Phase 3 clinical trial show. The therapy’s durability in sustaining factor VIII production at levels necessary to…

Two weeks ago, my daughter helped me move a small refrigerator. I had the front end and she had the back, which was open and exposed various parts. When we arrived at our car, I noticed her finger was bleeding quite a bit. The cut appeared to be long but…

When I was in my early teens, a family member introduced me to the law of attraction. According to this “law,” thoughts determine the outcome of one’s life. Therefore, if your thoughts are positive, positive things will happen in your life. I was young and determined to try it…

Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but many lack both the knowledge necessary for an informed decision and confidence regarding this form of treatment, according to a questionnaire and interview-based study. Most of those interviewed were unaware that gene therapies are…

Immunizations are sometimes tricky when a child has a bleeding disorder. The shots can cause bruising and swelling at the injection site, or even a muscle bleed. Each person is different, but nevertheless, precautions must be taken. When my children needed immunizations, I always gave them a dose of…

When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…

With all of the talk about COVID-19 vaccinations being right around the corner, I cannot help but feel like I can finally breathe. Hopefully, at this time next year the world will look back on the year past and proclaim, “We are free at last!” Life may return to normal,…

Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients with hemophilia B, according to the therapies’ maker, uniQure. The company announced in a press release that…