Sickle Cell Disease

Time To Get Ready For Rare Disease Day 2021!
Member 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…
TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo
Clinical 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…
EMA Accepts MAA For Oxbryta To Treat Haemolytic Anaemia In Sickle Cell Disease
Clinical 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,…
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical 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…
THALIA App: The Skilled Digital Assistant of People with Thalassaemia and Sickle Cell Disease
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THALIA App: The Skilled Digital Assistant of People with Thalassaemia and Sickle Cell Disease

Committed to continuing efforts to develop useful tools for individuals with thalassaemia and haemoglobin disorders worldwide, TIF introduces a novel mobile health application for patients with thalassaemia and sickle cell…
TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020
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TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020

TIF is proudly organising the European Thalassaemia & Sickle Cell Disease Symposium, a joint patients’ and healthcare professionals’ three-day educational event which will take place online on 11 – 13 December…
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
Clinical News

What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?

There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in…
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
Clinical News

EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
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…
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