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Targeting Shared Molecular Etiologies to Accelerate Drug Development for Rare Diseases
Session Chair(s)
David Andrew Pearce, PhD
President of Innovation and Research/Chair International Rare Disease Research
Sanford Health, United States
Many rare diseases share the same underlying molecular etiology that are therapeutically targetable. Basket trial design based on molecular etiology enables more patients to gain access to clinical trials and accelerate identification of new therpies
Learning Objective : Recognize the potential of basket trial design in rare disease; Apply basket trial design in rare diseases with share molecular etiologies.
Speaker(s)
Shared Platforms Approaches/Technologies for Rare Diseases
Philip (P.J.) Brooks, PhD
National Center for Advancing Translational Sciences (NCATS), NIH, United States
Deputy Director, Division of Rare Diseases Research Innovation
Targeting Shared Molecular Etiologies to Accelerate Drug Development for Rare Diseases
Christina Waters, PhD, MBA
RARE Science, Inc., United States
Chief Executive Officer and Founder
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