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Tag archives : TIF News

Thalassaemia Prior and Consequent to COVID-19 Pandemic: The Perspective of Thalassaemia International Federation (TIF)

  Patients with haemoglobin disorders, particularly β-thalassaemia or sickle cell disease (SCD) or combined forms, on account of their underlying disease pathology and associated (iron load mainly in the case of thalassaemia) co-morbidities, are defined as high-risk individuals, prone to develop more severe complications from SARS-COV-2. Despite the fact that ...

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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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FDA Grants Orphan Drug Designation to Mitapivat for Treatment of Thalassaemia

Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class pyruvate kinase-R (PKR) activator Mitapivat for the treatment of patients with NTDT α- and β-thalassaemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety ...

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FDA Approves New Formulation for Ferriprox (Deferiprone) Twice-a-Day Tablets

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, when current chelation therapy is inadequate. The new formulation of ...

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The 1st TIF Webinar for Medical Specialists scheduled for 11 May

The 1st webinar for Medical Specialists organised in the context of TIF’s eThalEd online course has just arrived! Dr Antonio Piga, Professor of Paediatrics & Dean of the Medicine School at San Luigi University Hospital of the Turin University, will be the host of this introductory webinar entitled ”Access to New Therapies: Reblozyl”. ...

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