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Rapamycin

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 Rapamycin, a drug widely used for almost 20 years to protect organ transplant patients, has been found to reduce, in mice, the build up of toxic proteins that destroy ...

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Mitapivat (AG-348)

  Update: 31 January 2020 The clinical proof-of-concept has been established based on a preliminary analysis of the Phase 2 trial of Mitapivat (AG-348) in patients with non-transfusion-dependent thalassaemia announced at the 61st Annual Meeting of the American Society of Haematology. The Phase 2 study has enrolled 12 of the ...

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Sickle Cell Disease

ARU-1801 Gene Therapy (SCD)   Update: 31 January 2020 The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, an experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassaemia. This designation means eligibility for a  priority review voucher once the FDA approves a biologics ...

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Apotransferrin

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019  Apotransferrinhas a physiological role in the transportation and distribution of iron among the ...

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