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.
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Hemophilia A is a genetic bleeding disorder caused by mutations in the *F8* gene, which leads to a deficiency in clotting factor VIII. Treatments include replacement therapies like SCT800 and Wilate. Experimental gene therapies, such as SPK-8016, have also been investigated.
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SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…
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).
Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that's approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.
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…
Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…
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.
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.