A natural anticoagulant protein called tissue factor pathway inhibitor (TFPI) is critical for defining the bleeding severity profiles of people with hemophilia A and B, researchers in France report. This discovery, which was found not to hold for healthy individuals, adds support to TFPI as an important element…

Chugai Pharmaceutical recently published data of a Phase I clinical study on the antibody emicizumab. The study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” was published in The New England Journal of Medicine. The study revealed possible benefits from emicizumab  for the prevention of bleeding in…

Rare Disease Day, observed this year on Feb. 29, serves as a poignant reminder of the challenges facing those who live with uncommon medical conditions. According to the event’s website, a rare disease is defined as one that affects fewer than 1 in 2,000 individuals. Among these conditions…

Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing. The move is backed by preclinical studies in nonhuman primates that show the treatment led to…

https://www.youtube.com/watch?v=z3f8elOVOrU This children’s video from Centers for Disease Control and Prevention focuses on how to tell your friends that you have hemophilia. MORE: How kids can play safe with hemophilia.  Billy, a young boy with hemophilia, answers some of the questions that his friends ask about the disease. He…

Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery. Esperoct received similar approval…

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research. The study, “Nongenotoxic antibody-drug conjugate conditioning…

As a person with von Willebrand disease (VWD) and hemophilia, bruises have been a near-constant presence in my life. VWD and hemophilia are bleeding disorders that affect the body’s ability to form blood clots and stop bleeding. Both can cause spontaneous and excessive bleeding, including bruising, heavy…

I have fond memories of my dad sitting at the kitchen table on Sunday mornings. After attending church, he read the newspaper while the radio played his favorite Tejano music. He always read articles to my mom as they sat together, enjoying the morning. The crisp newspaper would buckle as…

The vast majority of people in the hemophilia community support the implementation of newborn screening for the bleeding disorder in the United Kingdom, a study reports. The study, “Newborn screening for haemophilia: The views of families and adults living with haemophilia in the UK,” was published in…