The primary endpoint will evaluate the mean ratio of the Actual Fraction of Cells Remaining (FCRa; as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as predicted by the Spectra Optia system FCR algorithm), in the Evaluable subject population. The trial started in November 2012 and the estimated completion date is May 2013.


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This study, sponsored by Novartis Pharmaceuticals, aims to assess the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 24 and 52 weeks of treatment. Also, to evaluate the impact of deferasirox on the Quality of Life for adults and pediatric patients; correlate changes in serum ferritin and LIC, evaluate efficacy changes according to genotype, evaluate higher doses of deferasirox, assess endocrine function and examine pk parameters.


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bluebird bio

Gene therapy firm Bluebird Bio awarded $9.3 million from the California Institute for Regenerative Medicine (CIRM) to support a Phase I/II study evaluating bluebird’s LentiGlobin® treatment for β-thalassemia. The trial is scheduled to start in the U.S. next year. The CIRM grant is one of the first to have been made through the Institute’s new Strategic Partnership Awards initiative.


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children hospital philadelphia

The combination of deferoxamine and deferiprone has been shown to be particularly beneficial for reducing cardiac iron, but it requires a painful injection/infusion, which hinders adherence. This pilot study aims to investigate the safety of an oral-only combination chelator regimen (deferasirox and deferiprone) in individuals with thalassemia major with poorly controlled iron overload and to assess how well this chelator combination lowers iron stores over one year.


This study is sponsored by the Children's Hospital of Philadelphia in collaboration with the Cooley’s Anemia Foundation and the Toronto General Hospital.


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Sultan Bin Khalifa International Thalassemia Award

H.H. Dr. Sheikh Sultan Bin Khalifa Al Nahyan, the son and also the current Advisor for the President of the United Arab Emirates, has been deeply committed for the welfare of Thalassemia Community since a long time, having initiated several magnanimous projects for the benefit of Thalassemia Patients and their families.


As an extension of his vision, H.H. Dr. Sheikh Sultan Bin Khalifa Al Nahyan has now taken another noble initiative in recognizing these individuals and group efforts, for first time in the World, by establishing Sultan Bin Khalifa International Thalassemia Award in partnership with Thalassemia International Federation.


For more information, please visit the official website.