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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…
TIF’s Point of View: Medicines Regulatory Systems – Continued Strengthening Is Required
News

TIF’s Point of View: Medicines Regulatory Systems – Continued Strengthening Is Required

Access to quality-assured medical products improves health and saves lives. Nevertheless, one third of the world’s population lacks timely access to quality-assured medicines while estimates indicate that at least 10%…
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,…
TIF Updated Vaccinations & Therapeutic Drugs COVID-19 Guide Has Landed
News

TIF Updated Vaccinations & Therapeutic Drugs COVID-19 Guide Has Landed

A comprehensive Guide for both existing and developing vaccines and treatments against COVID-19, exclusively developed by TIF for its global thalassaemia community. Featuring: Pfizer, Moderna, AstraZeneca: What are the differencies…
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…
Blood Supply Challenges & Transfusion Care of Patients With Haemoglobinopathies During COVID-19 Pandemic
News

Blood Supply Challenges & Transfusion Care of Patients With Haemoglobinopathies During COVID-19 Pandemic

The COVID-19 pandemic has had an impact on blood supply in several countries, jeopardizing the already demanding and often suboptimal care for haemoglobinopathies patients, mainly in developing and low-income countries…
EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
News

EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia

The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia. This marks the first…
Just Updated: TIF’s Vaccinations & Therapeutic Drugs for COVID-19 Guide
News

Just Updated: TIF’s Vaccinations & Therapeutic Drugs for COVID-19 Guide

Everything you need to know about the different COVID-19 vaccines, both already available and developing, in one place! Discover our newly updated Guide ”Vaccinations & Therapeutic Drugs for COVID-19”, containing…
COVID-19 Vaccines and Haemoglobin Disorders: The Latest Position Statement of the Thalassaemia International Federation
News

COVID-19 Vaccines and Haemoglobin Disorders: The Latest Position Statement of the Thalassaemia International Federation

In view of the plans made by national health authorities in every country across the world for the widespread vaccination of individuals against the SARS-CoV-2 virus, the Thalassaemia International Federation…
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