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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 />

• Numerous scientific clinical research<br />

challenges<br />

<br />

<br />

<br />

Multiple organ involvement requires<br />

participation by many specialties that have<br />

not historically worked together<br />

Clinical focus shifts across the lifespan<br />

Meaningful endpoints <strong>of</strong> some clinical trials<br />

may take decades to determine

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