Research on rare diseases can shed light on mechanisms and therapeutic opportunities for common diseases. Better tools and improved support models have helped mitigate the special risks of research for rare diseases. This panel aims to educate everyone along the continuum from basic research through clinical trials about the infrastructure and innovation needed to accelerate the development of therapies for people with rare diseases.

Learning Objectives:

• Examine rare diseases research that has yielded broader drug discovery knowledge
• Discuss strategies to overcome barriers in developing therapies for rare diseases
• Highlight industry-changing innovations from rare diseases drug discovery and development

Ability Level: All

Session ID: 2004