Sickle cell
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أخبار
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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FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD
FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status…
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FDA Approves Oxbryta™ (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease
Global Blood Therapeutics, Inc. announced only a few hours ago that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta™ (previously known as Voxelotor) tablets…
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Hypertension Medication Valsartan May Help Prevent Vaso-Occlusive Episodes in Sickle Cell Disease
A medication used to treat high blood pressure, called Valsartan, may help decrease the stickiness of red blood cells and reduce the risk of vaso-occlusive crisis in people with sickle cell…
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TIF Publications
Beta Thalassaemia, Alpha Thalassaemia and Sickle Cell Disease Educational Community booklet (2014)
Eleftheriou A, Angastiniotis M English Greek
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TIF Publications
