Marisa Wexler, MS, senior science writer —

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to advance gene therapies for hemophilia and other genetic disorders. In CHOP’s 166-year history, this award — to recognize the most significant achievements in improving the health of children —…

Roche has extended its partnership with the World Federation of Hemophilia (WFH) to expand access to treatments for hemophilia through the end of 2028, the company announced. Under the partnership, Roche will continue to provide its prophylactic (preventive) treatment for hemophilia A to the WFH’s Humanitarian…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Osteoporosis and other bone disorders are more common in hemophilia carriers than in the general population, according to an analysis of U.S. insurance data. Its researchers also found a higher rate of bone problems among people with von Willebrand disease (VWD), another inherited bleeding disorder. “This study highlights the need…

Treatment with the experimental gene therapy Roctavian (valoctocogene roxaparvovec) significantly increased factor VIII levels in men with severe hemophilia A treated in the Phase 3 GENEr8-1 trial and with reasonable safety, findings at one and two years post-treatment show. While declines in these levels were evident over time,…

The first participant has been dosed in a Phase 1/2 dose-confirmation trial that is testing FLT180a, an experimental gene therapy that Freeline is developing to treat hemophilia B. “We are very excited about this progress in our hemophilia B program as our enhanced focus on execution is starting to…

The vast majority of women who have a bleeding disorder or are hemophilia carriers experience noticeable symptoms, such as bruising or heavy periods, a study based on survey results reported. These women also often mentioned encountering healthcare professionals who were dismissive or lacking in empathy. “Many of the experiences…

People with hemophilia may be less likely to develop cardiovascular disease (CVD) — disorders of the heart and blood vessels — according to a study from the U.K. and the Netherlands. “A lower-than-predicted [cardiovascular disease] incidence was found, supporting the theory that hemophilia protects against CVD,” the researchers wrote.

Nearly all men with moderate-to-severe hemophilia B given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have stopped using prophylactic therapies, full study results show. Trial data also indicated that EtranaDez, formerly known as AMT-061, is effective in people with antibodies against the viral vector…