Home » Author Archives: Catherine Skari (page 8)

Author Archives: Catherine Skari

Denosumab

  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 of denosumab for use in osteoporosis. Sources: https://www.novartis.com/news/media-releases/sandoz-announces-first-patient-enrolled-clinical-study-proposed-biosimilar-denosumab-osteoporosis ...

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Luspatercept

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 25 November 2019 The U.S. Food and Drug Administration (FDA) has approved REBLOZYL®(luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. The first and only FDA-approved erythroid maturation agent, ...

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Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 25 November 2019 Manufacturing specifications have been refined in order to aim for the first ‘commercial’ patient to undergo treatment in early 2020. Designation of qualified centres in the EU, signing paperwork and training is underway and will be announced ...

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Gene Editing

CRISPR gene editing   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 the first quarter of 2019 and data for ...

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Hepcidin

Hepcidin trials – TRANSCEND   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 is a hormone that performs various functions related ...

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Improving safety of blood transfusion

    INTERCEPT-treated RBCs   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 SPARC study was a randomized, double blinded cross-over ...

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Mr Abdul Baset Mohd Merdas

Mr. Abdel Basset Mohamed Merdas was born in the United Arab Emirates in Dubai in 1960, and holds a Bachelor Degree in Business Administration. He worked for the Dubai Government from 1989 until 2006. He is one of the founders of the Emirates Thalassemia Society (ETS), and played a major ...

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