Patients

On February 12, in an appraisal consultation document, the UK National Institute for Health and Care Excellence (NICE) recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) gene therapy for the…

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…

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…

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%…

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,…

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…

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…

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…

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…

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…