The Treatment of Thalassemia -- Slow Progress and New Dilemmas

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Abstract
The thalassemias are common single-gene disorders and pose an important public health problem in many countries. In recent years there has been great progress in determining the molecular pathology of the different forms of thalassemia and in understanding the basis for their remarkable clinical heterogeneity1. This knowledge has been applied in the clinic for the detection of carriers and the prenatal diagnosis of the more severe forms, particularly beta-thalassemia. The result has been a major reduction in the number of new cases of thalassemia, notably in Greece, Italy, and the Mediterranean islands2.Despite these successes, many children continue . . .