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Sickle Cell Disease Patients’ Views on Oxbryta

Sickle Cell Disease Patients’ Views on Oxbryta

OXBRYTA (voxelotor) is a prescription medicine used for the treatment of sickle cell disease (SCD) and constitutes the first aproved treatment that directly inhibits sickle hemoglobin polymerization, the root cause…
TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®

TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®

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…
Time To Get Ready For Rare Disease Day 2021!

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

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…
Meetings

Meetings

NOW PUBLISHED: The TIF ‘’Gene Therapy & Thalassaemia’’ Survey Report

NOW PUBLISHED: The TIF ‘’Gene Therapy & Thalassaemia’’ Survey Report

Gene therapy for thalassaemia is an expensive and complex procedure, with great efforts ongoing currently by all involved stakeholders, such as patients, healthcare professionals, etc. to ensure its accessibility and…
TIF’s Point of View: Medicines Regulatory Systems – Continued Strengthening Is Required

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%…
TIF Supports IAPO´s Call For The Ratification Of The African Medicines Agency Treaty

TIF Supports IAPO´s Call For The Ratification Of The African Medicines Agency Treaty

Two years after the Treaty to establish an African Medicines Agency was agreed, over 40 leading organizations, including the Thalassaemia International Federation (TIF), and hundreds of thousands of patients, researchers…
The Lancet: Russian Coronavirus Sputnik V Vaccine Achieves Over 90% Efficacy

The Lancet: Russian Coronavirus Sputnik V Vaccine Achieves Over 90% Efficacy

Russia’s Sputnik V vaccine was 91.6% effective in preventing people from developing Covid-19, according to peer-reviewed results from its late-stage clinical trial published yesterday in The Lancet international medical journal.…
EMA Accepts MAA For Oxbryta To Treat Haemolytic Anaemia In Sickle Cell Disease

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,…
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