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

myBIO Chatter

Speakers (4)

contact_8173 Robert Beall. PhD President and CEO Cystic Fibrosis Foundation
contact_7037 Michael Diem Director Business Development, Rare Diseases Unit GlaxoSmithKline plc
contact_8170 Jose Carlos Gutierrez-Ramos SVP, Head of BioTherapeutics R&D Pfizer Inc.
contact_4655 John Mendlein, Ph.D. Executive Chairman and CEO aTyr Pharma, Inc.

Moderators (1)

contact_3868 Rogerio Vivaldi SVP and Head of Rare Diseases Business Unit Genzyme, a Sanofi Company