Surveys

Gene Therapy & Thalassaemia

Gene Therapy & Thalassaemia

 Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells.   One of the novel…
Gene Therapy and Thalassaemia: The Patients‘ Perspective survey!

Gene Therapy and Thalassaemia: The Patients‘ Perspective survey!

  A significant number of gene therapy approaches are currently undergoing clinical trials, some of which are already producing encouraging results. The Bluebird Bio’s gene therapy product, under the name ZYNTEGLO™,…
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