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…

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…

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…

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…

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…

A panel of multidisciplinary experts have developed recommendations for applying best practice guidelines in the planning and managing of surgery in hemophilia patients. The guidelines were outlined in the consensus review, “Recommendations on multidisciplinary management of elective surgery in people with haemophilia,” published in the journal…

Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…