Clinical Trial Updates (SCD)

  • Ferriprox® (deferiprone)

      Update: 31 March 2023 No update available.   Update: 20 December 2022 No update available.   Update: 30 September 2022 No update available.   Update: 30 June 2022 No…

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  • Gene Therapy

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  • Gene Editing

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  • L-Glutamine

      Update: 31 March 2023 No update available.   Update: 20 December 2022 – The Qatar Ministry of Public Health has granted marketing authorization for the commercial distribution and sale…

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  • Mitapivat

      Update: 31 March 2023 Completed enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in adults with sickle cell disease. Anticipate to announce data and…

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  • FT-4202

      Update: 31 March 2023 No update available.   Update: 20 December 2022 No update available.   Update: 30 September 2022 No update available.   Update: 30 June 2022 The…

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  • FTX-6058

      Update: 31 March 2023 FTX-6058 is a small molecule designed to increase fetal haemoglobin. FTX-6058 is being developed for the treatment of sickle cell disease (SCD) and other hemoglobinopathies.…

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  • Adakveo (crizanlizumab)

      Update: 31 March 2023 Preliminary results from the ongoing global Phase III STAND study in sickle cell disease showed no difference between Adakveo and placebo in annual rates of…

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  • Oxbryta (voxelotor)

      Update: 31 March 2023 Preliminary draft guidance from England’s HTA body NICE states the product is not an acceptable use of National Health Service resources. Previously the UK regulatory…

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