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download pdf - Department of Pediatrics - University of Miami

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Addressing the Clinical and Translational<br />

Research Needs in SCD is not Easy!<br />

• Today, basic science supporting clinical and<br />

translational research in SCD is minimal<br />

<br />

<br />

NIH budget for SCD-related basic research is<br />

substantially smaller than for other low incidence<br />

diseases<br />

Pharmaceutical industry development <strong>of</strong> new<br />

orphan drugs has not been a priority, but that is<br />

changing

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