β-Τhalassaemia
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News
EU APPROVES MITAPIVAT | A New Treatment Milestone for Adults with α- or β-Thalassaemia
This decision is of particular importance because it covers a broad adult thalassaemia population, including both alpha- and beta-thalassaemia and both transfusion-dependent and non-transfusion-dependent disease. According to Agios Pharmaceuticals’ official…
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News
JUST RELEASED | 5th Edition of TIF’s Guidelines for the Management of Transfusion Dependent β-Thalassaemia
This edition stands as a seminal resource that provides critical guidance and recommendations to healthcare professionals across various specialties, researchers and all stakeholders involved in the care of individuals affected…
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Scientific News
CASGEVY | 1st Gene-Editing Therapy for Thalassaemia and SCD Approved in UK
Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The therapy, developed by…
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News
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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