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Publications

Denosumab

  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 Sandoz, a Novartis division focusing on biosimilars have enrolled the first patient in an intergrated Phase I/III study for the development ...

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Reblozyl

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 31 January 2020 Data evaluating Reblozyl® (ex-Luspatercept-aamt) were presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida. 1% of patients (101/224 patients receiving Luspatercept-aamt in the phase 3 BELIEVE study) achieved at least a 33% reduction ...

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Concluded/Terminated Studies-Trials

Assessment of long-term safety of deferiprone in children (DEEP-3 Study)     Update: 20 December 2018 The study has concluded.   Update: 02 October 2018 The safety of deferiprone, either alone or in combination with deferoxamine, was evaluated in 297 patients from 16 hospitals in 6 Mediterranean countries, who had ...

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Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 31 January 2020 New data from ongoing studies of LentiGlobin™ gene therapy for β-thalassaemia (betibeglogene autotemcel) in pediatric, adolescent and adult patients who have transfusion-dependent β-thalassaemia (TDT), including results from the Phase 3 Northstar-3 (HGB-212) study in patients with a ...

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Gene Editing

CRISPR gene editing   Update: 31 January 2020 No update available   Update: 25 November 2019 CRISPR Therapeutics and Vertex Pharmaceuticals announced positive, interim data from the first two patients with severe haemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001. 1 patient with transfusion-dependent beta-thalassaemia (TDT) received CTX001 in ...

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Hepcidin

Hepcidin trials – TRANSCEND   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 No update available.   Update: 01 February 2019 Hepcidin ...

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Improving safety of blood transfusion

    INTERCEPT-treated RBCs   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 No update available.   Update: 01 February 2019 The ...

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