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Tag archives : Healthcare Professionals

Thalassaemia Prior and Consequent to COVID-19 Pandemic: The Perspective of Thalassaemia International Federation (TIF)

  Patients with haemoglobin disorders, particularly β-thalassaemia or sickle cell disease (SCD) or combined forms, on account of their underlying disease pathology and associated (iron load mainly in the case of thalassaemia) co-morbidities, are defined as high-risk individuals, prone to develop more severe complications from SARS-COV-2. Despite the fact that ...

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FDA Grants Orphan Drug Designation to Mitapivat for Treatment of Thalassaemia

Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class pyruvate kinase-R (PKR) activator Mitapivat for the treatment of patients with NTDT α- and β-thalassaemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety ...

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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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