The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…
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Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using concizumab as a prophylactic (preventive) treatment compared with those who weren’t on any prophylaxis. That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which…
A look at how FVIII/FIX deficiency contributes to variable disease severity and provides the biologic foundation for modern hemophilia therapies.
Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.
Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy…
After a decade of living alongside my husband, Jared, and his severe hemophilia B, I’ve learned that not all bleeding is as straightforward as it may seem. Bleeds vary widely, not just in size but in severity and impact. What might look minor can be excruciating, while larger bleeds,…
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What Life With Hemophilia Means to These Patients
In this video from the Bleeding Disorders Community, hemophilia patients and their families talk about how the disease has affected their lives but more importantly, how they haven’t let the disease redefine who they are as people. MORE: Three tips for coping with a hemophilia…
Hemophilia B is a rare genetic bleeding disorder that impairs the blood’s ability to clot properly.
Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…
Inside hemophilia genetics, such as F8 and F9 mutations, inheritance patterns, and de novo variants. Plus, evolving approaches to females with hemophilia.