UniQure N.V.
Nov 25, 2019
How Gene therapy is changing the Beta-thalassemia Treatment outlook?
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-transfus...
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