CSL Behring, a global biotherapeutics company focused on the development and delivery of innovative therapies that are used to treat coagulation disorders, announced this week that the European Commission has approved Idelvion (albutrepenonacog alfa), its long-acting albumin fusion protein for the treatment of bleeding in patients with hemophilia B…

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…

What would you rather do: have a day to relax all to yourself, or eat whatever you want and not gain weight? Many of us have seen memes asking similar questions. To be honest, until this summer, I would occasionally answer them without much thought. But after facing struggles with…

When fellow columnist G Shellye Horowitz asked me to travel to Houston to support her through surgery for superior mesenteric artery syndrome, I didn’t hesitate. I knew I needed to be there. She told me I was the perfect choice because I understand hemophilia, its treatment,…

I surprised myself when I was in Montreal for a hemophilia conference in 2022. I decided to stay an extra day after the event to explore the city. I have to say, I found the most amazing gluten-free French pastry shop! The croissants and pain au chocolat were to…

I really hate asking for help. I have several theories why, but mostly, it boils down to a fear of inconveniencing others. Perhaps there are deeper causes, like needing to learn that I am worthy of accepting it and understanding that everyone deserves to have their needs met, both physically…