Genetic Disorders

Beta thalassemia, globally affects almost 288,000 people, with an incidence of 60,000 cases every year. A rare monogenic genetic disease, beta-thalassemia is caused by more than 200 mutations of the beta-globin gene (HBB). The Beta-thalassemia pharmacological treatment mainly comprises of regular blood-transfusion therapy. The blood transfusion mainly helps in suppressing the enlargement of erythroid. However, many times they cause a gradual accumulation of iron due to insufficient excretory function. Since the body is incapable to get rid of excess iron, thus iron binders (chelators) are used. Iron chelation therapies Some of the iron chelation therapies ...