Sickle cell

Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Clinical News

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…
Coronavirus Disease among Persons with Sickle Cell Disease
Clinical News

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…
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Clinical News

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…
COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
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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…
COVID-19 & Haemoglobin Disorders: Compilation of Information for Sickle Cell Disease (2020)
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.
COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations (2020)
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.
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…
FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD
Clinical News

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…
FDA Approves Oxbryta™ (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease
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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…
Hypertension Medication Valsartan May Help Prevent Vaso-Occlusive Episodes in Sickle Cell Disease
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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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