Hemophilia B patients living in urban mainland China have longer hospitalizations and significantly higher medical costs — including more expensive fees for coagulation factors concentrates — compared with patients with hemophilia A, a retrospective study shows. In fact, people with hemophilia B had inpatient medical costs more than…

Researchers have found that adults with severe hemophilia A coupled with pre-existing joint disease benefit most from prophylaxis to improve function, quality of life, and activities, and to better manage pain. The study, “Effect of late prophylaxis in hemophilia on joint status: a randomized trial,” was published in…

Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016 World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…

A single dose of the experimental gene therapy AMT-180 continues to be well-tolerated and to enhance blood clotting activity independent of factor VIII levels in mouse and primate models of hemophilia A, a study shows. Titled, “Human Dose Prediction of a Novel Factor IX Variant Gene Therapy…

For physically active men with severe hemophilia A, switching from their standard preventive treatment to Hemlibra (emicizumab-kxwh) can help reduce bleeding, a recent study suggests. This change not only improved their quality of life but also reduced the burden of treatment, allowing them to maintain their active lives.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…

Hemlibra (emicizumab-kxwh) safely supports joint health in children with hemophilia A without inhibitors through three years of treatment, according to interim data from a post-marketing study in Japan. The ongoing study, AOZORA (jRCT1080224629), is evaluating the long-term safety of Hemlibra and its impact on joint health in children ages…

The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…

Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…

People with hemophilia A who start on preventive treatment in the first years of life generally report better health-related quality of life and joint health, a study reports. “Our results indicate that delayed start of prophylaxis in an older cohort with severe [hemophilia A] can still achieve excellent bleeding…