Margarida Maia, PhD,  science writer—

Individually tailored prophylaxis, or preventive treatment, with Jivi (damoctocog alfa pegol) nearly halved the number of annual bleeding episodes in people with severe hemophilia A who had previously been on another replacement therapy, according to a Phase 4 post-marketing study. Researchers also observed that none of the patients…

The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…

SerpinPC, an experimental treatment being developed by Centessa Therapeutics, continues to be safe when used for up to nearly three years and to almost zero out the number of bleeds in adults with severe hemophilia A or B. Those findings come from a one-year extension of the…

GS1191, a gene therapy in the pipeline of Gritgen Therapeutics, increased factor VIII (FVIII) activity in the blood of people with hemophilia A taking part in a small clinical trial in China, resulting in fewer bleeding episodes. The fully enrolled investigator-initiated trial (ChiCTR2300073179) is testing the safety and…

People with hemophilia are living longer, but Black males still are more likely to die from the disease, and at a younger age, compared with white people, a U.S. study finds. More research is needed to understand the reason for this difference and to improve care and survival rates…

Switching from on-demand treatment to regular preventive therapy with extended half-life products may mean fewer bleeds, healthier joints, and better quality of life for people with hemophilia. That’s according to a new analysis of Phase 3 study data from trials involving more than 100 hemophilia patients that spanned a…

More bleeding episodes may mean a lower quality of life and less productivity at work for men with hemophilia A living in Europe, according to an analysis of data from the CHESS II retrospective study. The analysis, “The impact of bleeding event frequency on health-related quality of life…

Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…

A researcher at the University at Buffalo has won a three-year grant worth $1.5 million to develop a way to tame a patient’s immune system and prevent it from rejecting factor VIII as a replacement therapy for hemophilia A. “My interest in hemophilia started when I read a mother’s…

Hemophilia treatments in Thailand are limited due to a lack of budgetary resources, the high cost of medications, and an infrastructure inadequate for supporting patient care, a study found. These hurdles mean that people with hemophilia in the Southeast Asian country may not be able to access the care…