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Correspondence
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Volume 329:364 July 29, 1993 Number 5
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Therapy for Globin-Chain Disorders

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To the Editor: The clinical evaluation of treatments for disorders of globin-chain synthesis1,2,3,4 has entered an exciting new era. Decisions about the design and application of future trials will dramatically influence the rapidity of the advance against sickle cell disease and beta-thalassemia. An expanding list of experimental treatments for sickle cell disease includes hydroxyurea, erythropoietin, butyrate, and bone marrow transplantation. There is a real danger, however, that in five years we will still know little about which, if any, of these approaches is best suited to a particular patient. It is difficult to enroll sufficient numbers of patients in adequate . . . [Full Text of this Article]

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