Sickle cell
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Actualités
COMING UP: New TIF Webinar On »COVID-19 Vaccinations & Patients With Haemoglobin Disorders »
TIF is delighted to inform you that a Special Webinar on ‘’COVID-19 Vaccinations & Patients with Haemoglobin Disorders’’ will take place on Wednesday, 31 March 2021, at 14:30-16:00 EEST/12:30-14:00 GMT.…
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Actualités
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
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Actualités
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
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Actualités
Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…
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Actualités
Coronavirus Disease among Persons with Sickle Cell Disease
Individuals with sickle cell disease (SCD) are a particularly vulnerable group of patients, with a higher risk of severe complications due to COVID-19 infection than the general public. A recent…
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Actualités
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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Actualités
COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
An Expert Panel of Six Medical Professionals from various countries, such as India, the UK, Italy and Saudi Arabia, shared their experiences with TIF on the impact of the COVID-19…
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TIF Publications
COVID-19 & Haemoglobin Disorders: Compilation of Information for Sickle Cell Disease (2020)
This document, prepared by the Thalassaemia International Federation , is a compilation of information exclusively for Sickle Cell Disease patients with regard to the coronavirus pandemic. English
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Publications
COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations (2020)
A TIF-compiled document aiming to provide a division of risk levels for thalassaemia and SCD patients with regards to the ongoing COVID-19 pandemic. English Greek
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Actualités
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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