SCD

TIF Is Actively Participating In The 26th EHA Annual Congress
Clinical News

TIF Is Actively Participating In The 26th EHA Annual Congress

TIF is excited to be participating in the 26th EHA Annual Congress, held this year virtually on 09 – 17 June 2021. Dr Androulla Eleftheriou, TIF Executive Director, and Ms…
Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
Clinical News

Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies

bluebird bio, Inc. today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin…
Oxbryta Increased Haemoglobin And Reduced Haemolysis in Adults and Adolescents With SCD
Clinical News

Oxbryta Increased Haemoglobin And Reduced Haemolysis in Adults and Adolescents With SCD

Global Blood Therapeutics, Inc. announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study of Oxbryta® (voxelotor) tablets in patients with sickle cell disease…
TIF Presents Its Newly Launched SCD Educational Course for Healthcare Professionals On 9 April
News

TIF Presents Its Newly Launched SCD Educational Course for Healthcare Professionals On 9 April

TIF is delighted to announce the organisation of a Virtual Presentation Event of the ‘TIF Sickle Cell Disease Educational Course for Healthcare Professionals’ – an online course providing comprehensive information…
COMING UP: New TIF Webinar On “COVID-19 Vaccinations & Patients With Haemoglobin Disorders“
News

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.…
Time To Get Ready For Rare Disease Day 2021!
Member News

Time To Get Ready For Rare Disease Day 2021!

TIF joins forces with 300 million people around the world for the global observance of Rare Disease Day, coming up on February 28th. Rare Disease Day is an annual awareness…
TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo
Clinical News

TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo

The Thalassaemia International Federation (TIF) has been informed through official communication by both the European Medicines Agency (EMA) and bluebird bio that the latter has placed Phase 1/2 and Phase…
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
News

Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
Clinical 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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