Home » Author Archives: Catherine Skari

Author Archives: Catherine Skari

New and Updated Data on Key Thalassaemia & SCD Pipeline Therapies at the American Society of Hematology (ASH) 2019 Annual Meeting – 7-10 December 2019

  Celgene Corporation, Acceleron Pharma, Sangamo Therapeutics & Agios Pharmaceuticals are expected to present new and updated data on the progress of key ongoing therapies for thalassaemia and sickle cell disease (SCD) in the 61st American Society of Hematology Annual Meeting to take place in Orlando, Florida, 7-10 December 2019. ...

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Deferasirox Mylan: A new generic drug for the treatment of iron overload in β-thalassaemia major obtained approval by the EMA

  The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product “Deferasirox Mylan“, intended for the treatment of chronic iron overload due to blood transfusions in patients with β-thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias. The manufacturer of this medicinal product is Mylan S.A.S. “Deferasirox Mylan“ will be ...

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US government & Gates Foundation pledge $200 million to bring gene therapies to patients with Sickle Cell Disease & HIV

  The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale. The collaboration between the NIH and the Gates Foundation sets ...

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As survival increases in β-thalassemia, renal complications emerge

  In a scientific paper recently published in Hematology, researchers discussed the most common pathophysiologic and clinical manifestations of renal disease in patients with beta thalassaemia. “In recent years, the life expectancy of patients with thalassaemia has increased, and this has allowed previously unrecognized renal complications to reveal themselves,” said ...

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The future of genome editing in Europe – Bringing together the views of government, industry, patient communities, and civil society‘ representatives

  Genome editing is a breakthrough healthcare technology which has the potential to notably change treatment options, largely influence current healthcare models, and impact patients in clinical trials worldwide. The science is advancing rapidly, promising considerable increases in the efficacy and precision of genome editing products with potentially curative treatments ...

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Delegation visit to Oman – 10-11 September 2019

A TIF Delegation Visit to Oman took place on 10 – 11 September 2019 as a follow up to the 2017 visit which was conducted at the request of the Ministry of Health for the provision of technical expertise for the development of a comprehensive national control programme for thalassaemia ...

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