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Non-Profit Organizations Role in Accelerating Preclinical Compound Testing to Fuel the Rare Disease Clinical Pipeline
Session Chair(s)
Daniela Brunner, PhD
Founder
Science Forward, United States
A panel will review examples of preclinical drug screening platforms and consortia organized by non-profit organizations. Experts will present their experience and describe the challenges encountered, solutions found, and best practices defined.
Learning Objective : Discuss how rare disease non-profit organizations accelerate drug development; Develop consensus with industry, academia and patients; Identify unmet needs, optimize translational methods, criteria for compound selection, addressing gaps, ensuring quality, reproducibility, robustness and interpretation of data; Discuss challenges and solutions to transparency, data sharing and sustainability.
Speaker(s)
Tuberous Sclerosis Complex Preclinical Consortium
Dean Aguiar, PhD
Tuberous Sclerosis Alliance, United States
Director, Preclinical Research
Panelist
Salvatore La Rosa, PhD
Children's Tumor Foundation , United States
Chief Scientific Officer
Panelist
Dominique Pichard, MD
Rettsyndrome.org, United States
Chief Science Officer
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