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…

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…

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…

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…

Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…

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…

While cleaning up recently, I found a physician-authored exercise guide from 2002 for Filipinos with hemophilia. It was fascinating to read, especially knowing that at the time, synthetic factor products were still scarce here in the Philippines. Treatment options were limited, and many people with hemophilia still relied…

When people hear the word “hemophilia,” they often picture severe bleeding, bruising, or swollen joints. Those are the visible symptoms, the ones doctors, nurses, and even family members understand. But living with a bleeding disorder is so much more than what meets the eye. There are symptoms we…

Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes a median of almost three years after being treated, according to full data from the Phase 3 AFFINE clinical trial. These new data add to top-line…