What is Druggable New Therapeutic Targets in Rare Disease

Only a fraction of the more than 7,000 rare diseases identified have approved therapies available. Advances in DNA sequencing have led to the identification of the genetic cause of several rare diseases, which can potentially lead to a better understanding of the disease and development of targeted therapies. Hear from experts that have a pulse on new therapeutic targets in rare disease.

Speakers: Jason Sicklick, MD, Ye Liu, PhD