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Gene Therapy & Thalassaemia Version 2.3: A TIF Survey

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 therapies  for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion ...

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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™, has received positive comments from the scientific committees of the European drug regulatory authority (EMA). This means that the practical ...

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