Sickle Cell Disease
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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…
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Scientific Literature
Research in Sickle Cell Disease: From Bedside to Bench to Bedside
Sickle cell disease (SCD) is an exemplar of bidirectional translational research, starting with a remarkable astute observation of the abnormally shaped red blood cells that motivated decades of bench research…
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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…
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News
Hemanext® Inc. Receives CE Mark Certification For Innovative Red Blood Cell (RBC) Processing & Storage System
Hemanext Inc. announced today it has received a CE Certificate of Conformity for the CE Mark for its Hemanext ONE Red Blood Cell (RBC) Processing and Storage System, allowing the…
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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…
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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…
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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…
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Scientific Literature
Hydroxyurea Use Among Children With Sickle Cell Disease at King Abdulaziz University Hospital in Jeddah City
Background Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy that is very common and causes a great burden in Saudi Arabia and worldwide. This study aims to determine the…
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News
EMA Accepts MAA For Oxbryta To Treat Haemolytic Anaemia In Sickle Cell Disease
The European Medicines Agency (EMA) has accepted Global Blood Therapeutics (GBT) marketing authorisation application (MAA) for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia in sickle cell disease (SCD). According to the company,…
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News
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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