Hemophilia Gene Therapy has many potential benefits for patients with this disorder. However, this method has some drawbacks. First, it is not yet a licensed hemophilia treatment. It also has a high failure rate. Second, it causes severe liver damage in some patients. Third, it requires careful monitoring. Hemophilia Gene Therapy has a high failure rate, and it should be used only in cases where all other methods have failed.
Hemophilia is a disease with no cure. Patients with the disorder lack the key proteins necessary for clotting the blood. For example, hemophilia A patients lack the clotting factor VIII. In contrast, patients with hemophilia B have an enlarged clotting platelet area. This results in serious bleeds, joint damage, and other health issues. More than 60% of hemophilia patients have a severe form of the disease, with very low production of clotting proteins. Currently, the only approved hemophilia Gene Therapy is Hemlibra, which can be administered once a month. It has been shown that patients with hemophilia A can see a 50% reduction in FVIII activity over three years after the treatment.
Another promising approach is adeno-associated mediated gene transfer, which has shown promising results. Several clinical trials have reached phase III, which allows patients to stop receiving prophylactic therapy. This therapy can help patients overcome their hemophilia symptoms and avoid invasive procedures. It may even lead to a cure for the disease. The first phase of this new approach is now underway. The next step is to make the process of gene delivery as easy as possible for the patient. Current treatments for hemophilia A and B patients have been injected with replacement clotting factors, which raise their protein levels. Unfortunately, these injections must be repeated weekly, and are ineffective for some patients. Hemophilia C patients, meanwhile, can be treated with an antibody-drug that is given only once a month. Although this treatment is not effective for people with hemophilia A, it can still help the patient with the disease.
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