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Innovations and Research

As one of five thalassemia centers in the United States designated by the National Institutes of Health, the Children's Hospital Boston thalassemia program provides patients with access to experts in the field and the most cutting-edge clinical trials available in the world. One promising recent study, now awaiting approval by the FDA, tested the effectiveness of a new oral iron chelator, deferisirox.
Researchers from Children's Hospital Boston have been at the forefront of thalassemia research and treatment for decades. Important advances in the field that were made here include:

• Recognition of the important role of hypertransfusion
by Dr. David Nathan, Senior Associate in Hematology, in the 1960s.
• Demonstration of effective iron chelation by subcutaneous pump deferoxamine (Desferal ®)
by Dr. Richard Proper and David Nathan in the 1970s.
• Elucidation of the molecular biology of thalassemia syndromes
by Drs. Nathan, YW Kan, Stuart Orkin and others.
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