Margarida Maia, PhD, science writer —

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…

Female hemophilia A  patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…

Xoma closed a $9.6 million deal to buy from Aptevo Therapeutics the rights to receive a payment stream for sales of Ixinity (trenonacog alfa), an approved medication used to prevent and treat bleeding episodes in people with hemophilia B. Under the terms of the deal, Xoma will…

Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…