Bleeding Disorders
News
A new standard of care in hemophilia A?
Results of a phase 3 trial showed that prophylaxis with emicizumab significantly reduced bleeds, compared to no prophylaxis, in patients with...
From the Journals
Avatrombopag cut procedure-related transfusions in patients with thrombocytopenia, chronic liver disease
In both trials, the primary endpoint met a high degree of statistical significance.
News
FDA approves new factor VIII product for hemophilia A
Jivi is approved for on-demand treatment and as routine prophylaxis.
Latest News
Emicizumab beats factor VIII prophylaxis by a wide margin
In the HAVEN 3 trial, patients with hemophilia A without inhibitors had a significant reduction in bleeding events and no thrombotic events.
Feature
Marzeptacog alfa reduces bleeding episodes in hemophilia with inhibitors
Early results were presented at the Hemophilia Drug Development Summit in Boston.
Feature
Blood disorders researcher is finalist for Trailblazer Prize
Dr. Daniel Bauer of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, was recognized for his research on sickle cell disease and...
News
FDA grants priority review to drug for PNH
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for ALXN1210, a long-acting C5...
Conference Coverage
Integrated pain program reduced LOS for sickle cell patients
WASHINGTON – The small study is another data point showing the importance of multidisciplinary care.
News
Marzeptacog alfa may prevent bleeds in hemophilia A/B with inhibitors
BOSTON—The activated factor VIIa variant marzeptacog alfa has demonstrated efficacy as prophylaxis for patients with hemophilia A or B who also...
From the Journals
Feds aim to streamline gene therapy oversight
“It seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases,” Dr. Collins and Dr. Gottlieb wrote....
News
Health Canada approves emicizumab
Health Canada has approved emicizumab (Hemlibra®) for use as routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A patients...