CRISPR gene editing (SCD)   Update: 30 May 2019 No update available.   Update: 29 March 2019 The Phase 1/2 Study in Sickle Cell Disease (SCD) is an open-label trial aiming to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with ...

  Update: 30 May 2019  Apotransferrinhas a physiological role in the transportation and distribution of iron among the body organs. Apotrasferrin has received orphan drug designation from EMA A proof of concept study in the Netherlands will begin in the second half of 2019 lasting 16 weeks with a maximum ...

This is how this year’s voting results were shaped: Universal access to quality thalassaemia healthcare services: Building bridges with and for patients (Winning theme, 196 votes) Patients’ voice at the frontline: Advocating for a better future in thalassaemia care and management (112 votes) The globalization of thalassaemia – Common challenges ...

  This is how this year’s voting results were shaped: Universal access to quality thalassaemia healthcare services: Building bridges with and for patients (Winning theme, 196 votes) Patients’ voice at the frontline: Advocating for a better future in thalassaemia care and management (112 votes) The globalization of thalassaemia – Common ...

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VIT-2763   Update: 30 May 2019 No update available.    Update: 01 February 2019 A randomized, controlled, multi-centred phase 2 proof-of-concept trial will begin in the second half of 2019 to assess the effectiveness of the oral ferroportin inhibitor (VIT-2763) on iron overload in β-thalassaemia patients. Phase 1 trials have ...

  Update: 30 May 2019 No update available.   Update: 29 March 2019 SLN124 has been submitted to the UK Medicines and Healthcare Products Regulatory Agency (MHRA). Subject to approval from the MHRA, Silence intends to begin a Phase I/II First-in-Human study in ß -Thalassemia and MDS patients, with the ...

The epidemiology project of TIF focuses on the collection of information on carrier rates, anticipated births and current status of haemoglobinopathy control strategies within health systems in the different country members of TIF, for β- and α-thalassaemiaσ, HbE and Sickle Cell Disease (SCD), and combinations constitute the main focus of ...

This is one of TIF’s new projects undertaken for the year 2013 and its goal is to develop a database for Haemoglobinopathy reference centres across the world, mapping the services they provide to patients. This is an extension to the information compiled by ENERCA-3 project and the deliberation of EUCERD, ...