Clinical Trial Updates (SCD)

  • Ferriprox® (deferiprone)

    Update: 30 June 2022 No update available.   Update: 25 April 2022 No update available   Update: 10 January 2022 No update available.   Update: 27 October 2021 Regulators in…

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  • Gene Therapy

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  • Gene Editing

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  • L-Glutamine

    Update: 30 June 2022 No update available.   Update: 25 April 2022 No update available   Update: 10 January 2022 No update available.   Update: 27 October 2021 Submission of…

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  • Mitapivat

    Update: 30 June 2022 Initiated the sickle cell disease cohort of the Phase 1 study of AG-946. Aiming to complete enrollment in the Phase 2 portion of the RISE UP…

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  • FT-4202

    Update: 30 June 2022 The study description was presented at EHA2022: Sources: https://s3.eu-central-1.amazonaws.com/m anage.com.storage.eha/temp/eha22_abstract_bodies/PB2232.html.pdf   Update: 25 April 2022 Etavopivat Phase I trial has been completed with results (presented at…

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  • FTX-6058

    Update: 30 June 2022 No update available.   Update: 25 April 2022 No update available   Update: 10 January 2022 No update available.   Update: 27 October 2021 No update…

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  • Adakveo (crizanlizumab)

    Update: 30 June 2022 No update available.   Update: 25 April 2022 Adakveo (crizanlizumab) is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients…

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  • Oxbryta (voxelotor)

    Update: 30 June 2022 Presented at EHA2022: Data from the Retrospective Study to Evaluate Outcomes in Patients with Sickle Cell Disease Treated with Oxbryta (RETRO), the first multicenter, retrospective study to examine the real-world…

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