Thalassemia: a look to the future

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Abstract

To assess the future of a clinical science one must first assess the rate of accrual of understanding in the past century. From the time of Cooley's description (1925) to 1964, the year of the first Symposium, progress was glacial because the molecular biology revolution in medicine was in its infancy and therapy other than transfusion was impossible. But between 1964 and 2015, progress has been huge on every front. Our patients ushered in the molecular biology revolution in medicine. They have benefited from far better understanding of molecular pathophysiology, substantial improvements in transfusion and chelation, more effective stem cell transplantation, the beginnings of gene therapy, and now major advances in our capacity to reinduce fetal hemoglobin. We have only lagged in the application of prevention technology in the less developed world that suffers the most from thalassemia and sickle cell disease. We must redouble our efforts to spare patients from these cruel diseases.

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