A team led by Lurie Children’s Hospital of Chicago researchers reported a case study of a newborn with combined spontaneous splenic rupture and severe hemophilia A. This baby, whose case is believed to be the first documented, was successfully treated without surgical intervention. Results from the study, “Successful medical management of a…

Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…

The biotech companies Swedish Orphan Biovitrum AB (Sobi) and Biogen have announced that the European Commission (EC) has approved their extended half-life therapy Alprolix (rFIXFc) for hemophilia B in all 28 European Union (EU) countries. In addition, the EC maintained the drug’s orphan designation. The World Federation of Hemophilia (WFH) estimated…

Researchers at the Centre Hospitalier Metropole Savoie in France identified a rare case of associated myelofibrosis (MF) and acquired hemophilia A (AHA), which they believe to be only the second case reported to date. The study, “Myelofibrosis and acquired hemophilia A: a case report,” was published in the…

Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…

Last week, my wife, Cazandra, and I attended the National Bleeding Disorders Foundation’s (NBDF) annual symposium. As parents of two adult sons who have severe hemophilia A, or factor VIII deficiency, we’re familiar with the lay of the land. The conference provided a space for us to discover…

I walked into the bleeding disorders conference filled with anticipation. After living abroad for over a decade, I had returned to the U.S. and was excited to connect with experts and patients to learn more about managing hemophilia as a woman. As I entered the event, I was mesmerized…

Kids have a way of flattening the extraordinary into the ordinary. My 6-year-old daughter once scraped her knee, looked down at the blood, and calmly said, “Like Daddy, because he bleeds.” Then there was the day she spotted a cockroach flipped upside down, thrashing helplessly on the floor. Without hesitation,…

Delaying the start of immunosuppressive treatment in adults diagnosed with acquired hemophilia A who are receiving Hemlibra (emicizumab-kxwh) to prevent bleeding may reduce the risk of infections and extend survival, a clinical study found. Patients in the Phase 2 study (NCT04188639 received Hemlibra for 12 weeks while immunosuppression…