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 ...Read More »
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 ...Read More »
TIF to organize a Capacity Building Workshop for Patients’ Organizations and individuals with thalassaemia in October 2019!
This fall TIF is coming to Germany✈️✈️ and is more than excited to announce its forthcoming Capacity Building Workshop for Patients’ Associations and people with thalassaemia and other haemoglobinopathies, which will take place on 11-13 October, 2019 in Hamburg, Germany!!! For more details, please click hereRead More »
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 ...Read More »
A comprehensive article by Carsten W. Lederer (PhD), Petros Patsali (PhD), Panayiota Papasavva (MD, MSc), Thessalia Papasavva (PhD) and Marina Kleanthous (PhD, Head of the MGTD), on the thalassaemia carrier status of Cypriots, and the therapy developments in the country.Read More »
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 ...Read More »