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Author Archives: Catherine Skari

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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Delegation visit to Austria – 11-13 April 2019

  TIF’s Board of Directors Member and Expert Patient, Mr. George Constantinou, and TIF’s collaborators Dr. phil Eva-Maria Knoll and Dr. Anne Yardumian, were delegated to visit Austria on 11-13 April, 2019, a country of special focus for the THALIA project, due to the fact that it has become a ...

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Living with Transfusion-Dependent β-Thalassaemia (TDT)

In its most severe form and without adequate care, thalassaemia can be lethal in early childhood, and although  International Thalassaemia Day has come and gone, awareness is paramount.   Andrew Obenshain, Head of Europe at gene therapy company bluebird bio, looks into what it means to live with thalassaemia, with ...

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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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World Hemophilia Day 2019

World Hemophilia Day is an international awareness day for hemophilia and other bleeding disorders. It is held annually on April 17, date of the birthday of Frank Schnabel, founder of the World Federation of Hemophilia. Since 1989, World Hemophilia Day is the day the whole bleeding disorders community comes together ...

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