Rare Disease Clinical Trials Are They Measuring The Right Things

Summary

Erika Augustine, MD, of the University of Rochester talks about the need to have a dialog with the key players in the What are the regulatory considerations for designing a Timothy Craig, DO of the Penn State Milton S. Hershey The Ohio State University Center for This week on The CheckRare Brief, we are joined by Eugene Lee, Head of CMI Media Group's The Duke-Margolis Center for Health Policy, under a cooperative agreement with the FDA, convened this two-day event that will ... Recorded live at the 2026 ASGCT Annual Meeting in Boston. This video is a portion of the symposium, "Advancing Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children's Hospital at Montefiore, New York City, ... Join Dr. Bradley L. Schlaggar, president and CEO of Kennedy Krieger Institute, and Dr. Erika Augustine, associate chief science ... There are approximately 7000 known