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أرشيف الكاتب: tifwp

Ιmara

  Update: 29 March 2019 Developed for sickle cell disease, IMR-687 is a highly potent, selective inhibitor of phosphodiesterase-9 (PDE9i) in blood cells. It targets the same biochemical pathway as hydroxyurea, a chemotherapeutic agent, but without its safety issues. IMR-687 has been shown in cell and animal models to increase ...

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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: 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: 29 March 2019 The first of three cohorts has been successfully dosed in a phase I study to assess the safety of subcutaneous administration of Sevuparin for the treatment of SCD. This double-blind, placebo-controlled Phase I study, complements the currently ongoing Phase II study which is examining the ...

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SLN-124

  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 first patient expected to enter the study in ...

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