Symposium: Translating the Translational Sciences - Different Understanding, Common Aim 1

Moderator(s)

• 

  Xiaofei Wang, PhD

  Pharmacologist
  U.S. Food and Drug Administration
  Silver Spring, Maryland

Presentations:

• 9:00 AM – 9:30 AM MT

  Biomarkers' Role in Accelerated Approval for Rare Neurological Diseases
  

  Speaker: Vishnu Sharma, PhD (he/him/his) – Office of Clinical Pharmacology, Office of Translational Sciences, CDER/FDA
• 9:30 AM – 10:00 AM MT

  Opportunities for Model-Informed Drug Development and Biomarker Strategies in Rare Diseases
  

  Speaker: Sandra Visser, PhD (she/her/hers) – Independent Consultant Quantitative Medicine
• 10:00 AM – 10:30 AM MT

  Biomarkers of Rare Diseases for Cell and Gene Therapy Products
  

  Speaker: Million A. Tegenge, PhD (he/him/his) – Office of Clinical Evaluation, Office of Therapeutic Products, CBER/FDA
• 10:30 AM – 11:00 AM MT

  The Value And Application Of Biomarker Strategy To Rare Disease Drug Development
  

  Speaker: Rachael Hawtin, PhD (she/her/hers) – Ultragenyx