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Our mission is to provide healthcare professionals with unbiased clinical research information, easily.

Currently, you can access the following clinical trials being conducted worldwide:

379206 studies
in
220 countries
Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 06/23/2021.
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At Bolder Science, we want your clinical trial search experience to be the best it can be. Complete the following prompts to easily find the trials you are interested in and see trials recruiting near you. You can adjust these selections in your dashboard after creating your account.

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Our Science

Beta-thalassemia

Pathophysiology

Beta-thalassemia is an inherited red blood cell disorder caused by mutations in and around the??-globin gene and characterized by reduced or absent ?-globin chain synthesis, decreased hemoglobin in the blood, and reduced functional red blood cell production.1-4?Loss of ?-globin results in a toxic accumulation of the??-globin protein in developing erythrocytes, which leads to ineffective erythropoiesis and the development of moderate to severe anemia.2,3,5 Patients with beta-thalassemia major typically present with clinical symptoms by 2 years of age and require red blood cell transfusions to survive; however, regular transfusions?may?lead to?iron overload and?potentially?life-threatening complications.1,3,6

Landscape

The need for additional treatment options in beta-thalassemia remains high. Investigational therapies include erythroid maturation agents, gene therapy, and gene editing, which aims to correct the imbalance between ?-globin and ?-globin that is characteristic of beta-thalassemia.7
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The safety and efficacy of the agents and/or uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.

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Worldwide trials investigating in Beta-thalassemia

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