Innovative Therapies
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News
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease…
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News
Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges
TIF, in collaboration with Thalassemics India and Thalassemia Patients Advovacy Group – India, conducted on June 12, 2020 a Webinar on the novel treatments emerging for thalassaemia and the hopes…
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News
European Commission Approves Reblozyl for the Treatment of Transfusion-dependent β-Thalassaemia
Bristol Myers Squibb (BMS) & Acceleron Pharma announced today that the European Commission approved Reblozyl (luspatercept) for the treatment of Transfusion-Dependent β-#Thalassaemia (TDT) in adult patients. Reblozyl has the potential to…
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Surveys
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TIF News
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Webinars
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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TIF Publications
Explaining Gene Therapy in Thalassaemia (2020)
The second educational brochure from the TIF’s ‘Gene Therapy’ booklet series, providing an in-depth view of how this revolutionary therapy works in patients with β-thalassaemia major.
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News
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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News
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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