Home » Author Archives: Catherine Skari (page 5)

Author Archives: Catherine Skari

Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 30 May 2019 Upon receipt of marketing authorisation from the European Commission by the end of June, Bluebird expects to launch first in Germany, followed by Italy, France and the U.K. in 2020 Analysts estimate prices of $900,000 in Europe ...

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Gene Editing

CRISPR gene editing   Update: 30 May 2019 The FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion dependent beta thalassaemia. Sources: https://www.globenewswire.com/news-release/2019/04/16/1804601/0/en/CRISPR-Therapeutics-and-Vertex-Announce-FDA-Fast-Track-Designation-for-CTX001-for-the-Treatment-of-Beta-Thalassemia.html   Update: 29 March 2019 The Phase 1/2 Study in Beta Thalassaemia is an open-label trial aiming to assess the safety and efficacy ...

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Hepcidin

Hepcidin trials – TRANSCEND   Update: 30 May 2019 No update available.   Update: 01 February 2019 Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body.  Thalassaemia patients have low hepcidin levels due to iron overload. Abnormally low hepcidin levels caused by ...

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Improving safety of blood transfusion

    INTERCEPT-treated RBCs   Update: 30 May 2019 No update available.   Update: 01 February 2019 The SPARC study was a randomized, double blinded cross-over study evaluating the efficacy and safety of INTERCEPT RBC in chronic anaemia patients. A total of 86 thalassemia patients were enrolled at three participating ...

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