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Archives de cet auteur: tifwp

Sickle Cell Disease Update

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 ...

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Apotransferrin

  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 ...

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International Thalassaemia Day 2019 – Get inspired, get involved!

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 ...

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Ferroportin inhibitors

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 ...

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SLN-124

  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 ...

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International Thalassaemia Day 2019 – Get inspired, get involved!

  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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Centres de référence des hémoglobinopathies

C’est l’un des nouveaux projets de la FIT pour l’année 2013 dont l’objectif est de développer une base de données pour les centres de référence des hémoglobinopathies à travers le monde, en cartographiant les services qu’ils fournissent aux patients. Il s’agit d’une extension des informations compilées par le projet ENERCA-3 ...

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