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Author Archives: tifwp

Ferroportin inhibitors

  VIT-2763  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 shown positive results with a consistent lower serum ...

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Voxelotor

  Update: 01 February 2019 The preliminary results from phase IIa HOPE trials for the safety and efficacy of voxelotor in SCD were presented at the 2018 ASH Annual Meeting. Results reflected the findings of two randomized, multi-centred trials with 154 patients participating – one with adults and adolescents over ...

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Sevuparin

  Update: 01 February 2019 Sevuparin is a new compound based on the commonly used blood thinner, heparin. It is able to restore blood flow and prevent obstruction of small blood vessels due to its anti-adhesive and anti-inflammatory characteristics. Received Orphan Drug Designation by FDA and EMA. A multi-centre, phase ...

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SLN-124

  Update: 01 February 2019 Silence Therapeutics is working on the development of therapeutic RNA molecules that use the body’s natural mechanism of RNAi to stop (or silence) targeted / specific genes in the genome from producing disease-causing proteins (i.e. gene silencing). RNAi is the natural regulation of gene expression ...

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Epidemiology data bank on haemoglobin disorders

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 ...

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Haemoglobinopathy Reference Centres

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, ...

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