Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…

https://www.youtube.com/watch?v=sK2EIwmIYPo This video from the Bleeding Disorders Community focuses on sports for children with hemophilia. It explains that physical activity is important for people living with hemophilia, as it helps build muscle strength and maintain physical fitness, but that not all sports are suitable for those living…

https://www.youtube.com/watch?v=sK2EIwmIYPo This video from the Bleeding Disorders Community focuses on sports for children with hemophilia. It explains that physical activity is important for people living with hemophilia, as it helps build muscle strength and maintain physical fitness, but that not all sports are suitable for those living…

https://www.youtube.com/watch?v=sK2EIwmIYPo This video from the Bleeding Disorders Community focuses on sports for children with hemophilia. It explains that physical activity is important for people living with hemophilia, as it helps build muscle strength and maintain physical fitness, but that not all sports are suitable for those living…

https://www.youtube.com/watch?v=sK2EIwmIYPo This video from the Bleeding Disorders Community focuses on sports for children with hemophilia. It explains that physical activity is important for people living with hemophilia, as it helps build muscle strength and maintain physical fitness, but that not all sports are suitable for those living…

Complication rates for elective surgeries in people with inherited bleeding disorders like hemophilia are low in Australia, reflecting good safety in their performance despite guidelines not always being followed, a study reported. Use of a specialty hemophilia treatment center (HTC) for surgery, and use of factor replacement therapy were among guidelines sometimes…

Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…

Most people with hemophilia responding to a survey in the Netherlands were generally satisfied with their treatment care. Patients’ interactions with healthcare professionals, availability of care, and coordination were cited as the most important factors for satisfaction. However, they reported improvements were needed regarding information about new treatments, their…

In an interview with Genentech, 13-year-old Beau talks about what it’s like living with the bleeding disorder hemophilia. MORE: Seven recommended books to learn more about hemophilia Beau is just like other boys his age — in his spare time he plays computer games and is…

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…