Sickle Cell Disease

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
Clinical News

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Fulcrum Therapeutics, Inc.  a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and…
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…
TIF Takes Active Part At The IAPO 9th Global Patients Congress
NOVITÀ

TIF Takes Active Part At The IAPO 9th Global Patients Congress

The International Alliance of Patients’ Organizations (IAPO) conducted online the 9th Global Patients Congress #GPC2020 on 16-17 September 2020, under the theme ”Co-creation of Innovative Healthcare”. The Congress featured a…
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
NOVITÀ

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…
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…
IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
Clinical News

IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the…
COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
NOVITÀ

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.
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