A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, with a planned advisory committee meeting having been canceled. The U.S. Food and Drug Administration (FDA) had requested, but now no longer intends, to meet with…
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It’s not easy to live with hemophilia in the Philippines. When we hear stories of hemophilia care around the world, it’s hard not to feel envious of hemophiliacs outside of our country who receive proper healthcare and can live a normal life. In this column, I will tackle the issue…
Adynovate (antihemophilic factor, rurioctocog alfa pegol), sold as Adynovi in Europe, is a recombinant (lab-made) clotting factor VIII (FVIII) concentrate given as an intravenous (into-the-vein) injection to prevent and control bleeding in children and adults with hemophilia A.
Hemophilia B and experimental gene therapy have been in the news lately. I was even surprised to hear a story on the topic while listening to my local NPR station. Hemophilia BÂ is the less common form of the disorder that arises when a…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective…
Social Clips
How Kids Can Explain Hemophilia to Their Friends
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…
Gene therapy directly addresses hemophilia B’s underlying cause by providing a healthy version of the F9 gene. In this way, it is expected to offer lasting bleed control with minimal or possibly no need for regular preventive therapies.
The severity of a person’s hemophilia depends on the amount of clotting factor they have in their blood. Generally speaking, the disease is considered mild, moderate or severe, although around 70 percent of people living with the disease have the severe form. MORE:Â Five tips for healthy living with hemophilia…
Treatments with anti-coagulation drugs such as Brilinta (ticagrelor) may trigger acquired Hemophilia A (AHA) in adults. This finding was reported by Italian doctors after a patient on Brilinta was hospitalized with a hemorrhage. The study, “Acquired Hemophilia A May Be Associated with Ticagrelor Therapy in a 52-Year-Old Man After a…
When my first son, Julian, was born in 1996 and diagnosed with severe hemophilia A, I thought my world was ending. I expected to give birth to a healthy baby boy. I never dreamed he’d have a medical condition. I went through the stages of grief: denial,…