Innovative Therapies
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NOVITÀ
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Education
eThalED COURSE: Access to New Therapies – The Case of Reblozyl by Dr. Antonio Piga
The 1st webinar for Medical Specialists organised in the context of TIF’s eThalED online course is here. Dr Antonio Piga, Professor of Paediatrics & Dean of the Medicine School at…
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NOVITÀ
Accessibility & Affordability of Novel Therapies for Thalassaemia: The Patients’ Perspective
9 patients with thalassaemia, members of TIF’s Patient Advocacy Group for Thalassaemia and Sickle Cell Disease (T-PAG), are discussing with the Thalassaemia International Federation the accessibility and affordability of the…
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NOVITÀ
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle…
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TIF Publications
Gene Therapy in β-Thalassaemia and Other Haemoglobin Disorders (2019)
An educational leaflet aimed at improving the awareness and comprehension of people with thalassaemia and other haemoglobinopathies worldwide about gene therapy.
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