A 70-year-old man with severe hemophilia A was treated for COVID-19 while receiving prophylactic, or preventive, treatment with Hemlibra (emicizumab) to lower his bleeding risk. This case underscores the importance of close cooperation…

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

I am not neurotypical. My thoughts, perception, and speech can be different than others. It is subtle, but there. For example, if someone tells me something, I take their word literally — and I mean literally. If someone tells me they poked themselves 30…

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are…

Bayer’s Jivi safely and effectively prevents bleeds during and after minor surgery in children, adolescents, and adults with severe hemophilia A, according to interim data from the Phase 2/3 PROTECT VIII and the Phase 3 PROTECT VIII Kids clinical trials. Most of the included patients were on…

Preventive, or prophylactic, treatment with Hemlibra (emicizumab) safely and effectively managed bleeding in adults and children with severe hemophilia A, a real-world study has found. The study, “Emicizumab prophylaxis: Prospective longitudinal real‐world follow‐up and monitoring,” was published in the journal Haemophilia. Originally developed by Roche’s subsidiary…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…

A first patient has been dosed in XTEND-Kids, a Phase 3 clinical trial evaluating the safety and efficacy of the investigational replacement therapy efanesoctocog alfa in boys with severe hemophilia A. Hemophilia A is caused by the lack of a functional blood clotting protein called factor VIII…

Long-term treatment with Jivi safely and effectively reduced the number of bleeding episodes in adolescents and adults with severe hemophilia A, according to up to seven years of data from the Phase 2/3 PROTECT VIII trial and its extension study. The new data confirmed the previous findings from…

I live in the middle of nowhere. It is one of the most beautiful places on the planet. The forest and the ocean intersect, creating some of the most gorgeous hiking trails I have had the pleasure of traversing. Nature is our playground, with thousands of acres of beaches, forests,…