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TIF by the side of thalassaemia patients in Syria

  The World Health Organisation (WHO) has contacted the Thalassaemia International Federation (TIF) to request the provision of its expertise and technical support on the thalassaemia care and management of patients in the North West Syria region. Currently, this region includes four Thalassaemia Centres with approximately 500 patients. These patients ...

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FDA Grants Priority Review to Potential Sickle Cell Disease Treatment Voxelotor

  The Food and Drug Administration (FDA) will review an approval application for a drug designed to target the root cause of damage from sickle cell disease (SCD). South San Francisco, California-based Global Blood Therapeutics announced Thursday that the FDA had accepted its application seeking accelerated approval for Voxelotor, an oral drug ...

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TIF releases new video on thalassaemia prevention

  Thalassaemia is a hereditary blood disease that CAN be prevented! But in what ways? ΤΙF’s latest educational video shines a spotlight on the essential aspect of the disease’s prevention, still challenging  in numerous countries across the world. Our goal? To further improve understanding and knowledge of the prevention’s vital ...

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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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Gene Therapy for β-Thalassaemia Approaches European Market Approval

Gene therapy continues to evolve with new emerging treatments finally reaching its promised potential: providing a one-time lifelong cure for even the rarest and most severe of genetic disorders. TIF’s Expert Advisor, Dr. Carsten Werner Lederer, explores this critical issue in his article, by highlighting the recent conditional approval for ...

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