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Gene Therapy & Thalassaemia

Highlighting the Patients' Views on Gene Therapy for 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 therapies for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion dependent β-thalassaemia ZYNTEGLO by Bluebird Bio that gives patients the potential to reach complete transfusion independence.

As part of our advocacy efforts that aim to help facilitate and support patients’ access to Gene Therapy and other New and Innovative Therapies for the treatment of thalassaemia, we are conducting a quick online survey on gene therapy to gather and assess your own perspectives and views on this important matter.

  • How much do you know or understand about gene therapy?
  • What do you believe its benefits will be for patients?
  • Would you be willing to undergo gene therapy, if you were given such an option?

This survey has been completed!

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