BioMarin is considering pricing its hemophilia A gene therapy Valrox (valoctocogene roxaparvovec), should it be approved, at $2 million to $3 million. That range would make Valrox the world’s most expensive one-time therapy. But, the company argues, this first gene therapy for any form of inherited hemophilia could…
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Hemophilia A is a rare genetic disorder caused by a deficiency of clotting factor VIII, leading to prolonged bleeding. While there is no cure, treatments like prophylaxis, on-demand therapy, and emerging gene therapies help manage symptoms and prevent complications.
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7 Recommended Books to Learn More About Hemophilia
Hemophilia is a genetic disorder that prevents blood clotting. Most common in men, there are two main types of hemophilia: hemophilia A and B. Below is a curated list of recommended reads for family members, friends and carers of patients with hemophilia, with help from Good Reads and Alibris.
There is no cure for hemophilia A, but several treatments are available to help prevent and control bleeds, reduce the risk of complications, and improve quality of life. People with hemophilia A are susceptible to excessive and prolonged bleeding episodes because their bodies don’t make enough functional factor…
SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
Hemophilia is a disorder wherein the blood is not able to clot properly. As a result, patients bleed easily, and it's unusually prolonged and heavy.
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Explaining Hemophilia A and B
https://www.youtube.com/watch?v=S_YMGmHqIW4 This video from Claire Blatt shares a lecture about the effects of two blood-clotting disorders: hemophilia A and hemophilia B. MORE: A study shows that NovoSeven counters bleeding in children with hemophilia. Though the lecture is aimed at nurses, it’s helpful for anyone looking to learn about these…
While hemophilia A and hemophilia B are both bleeding disorders characterized by deficiencies in blood clotting factors, there are differences in one type versus the other, particularly in the specific genetic mutations that cause the disease, and in some of the treatment methods.