Home » Tag Archivi: Healthcare Professionals

Tag Archivi: Healthcare Professionals

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 ...

Leggi Articolo »

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. ...

Leggi Articolo »

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”. ...

Leggi Articolo »

TIF publishes new material on the COVID-10 Pandemic

TIF continues the efforts to address the questions and concerns of its global patients’ and healthcare professionals’ community about the COVID-19 Pandemic and, in doing so, it has issued 2 brand new informational documents. The TIF-proposed Haemoglobinopathy Patient Pathway for Day Care Centres, Units and Clinics treating patients with thalassaemia ...

Leggi Articolo »