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Tag archives : Featured

FDA Approves New Formulation for Ferriprox (Deferiprone) Twice-a-Day Tablets

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, when current chelation therapy is inadequate. The new formulation of ...

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FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD

CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle Cell Disease (SCD). As with the FDA’s breakthrough ...

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Reblozyl (luspatercept-aamt) Receives Positive CHMP Opinion for the Treatment of Adults with β-Thalassaemia

  On 30 April 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product REBLOZYL, intended for the treatment of adults with beta-thalassaemia or transfusion-dependent anaemia associated with myelodysplastic syndromes (MDS). The applicant for this medicinal product is Celgene Europe BV. ...

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The 1st TIF Webinar for Medical Specialists scheduled for 11 May

The 1st webinar for Medical Specialists organised in the context of TIF’s eThalEd online course has just arrived! Dr Antonio Piga, Professor of Paediatrics & Dean of the Medicine School at San Luigi University Hospital of the Turin University, will be the host of this introductory webinar entitled ”Access to New Therapies: Reblozyl”. ...

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Oxford Set To Start First UK Coronavirus Vaccine Trial Today

The first UK vaccine candidate to prevent coronavirus infection is set to begin clinical testing in human volunteers  today. Its developers at the University of Oxford hope to have 1m vaccine doses ready by September to carry out large-scale clinical trials during the autumn and, if all goes well, to ...

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