PEDIATRIC RARE DISEASE SYMPOSIUM
Time Topic Speaker
9:00 – 10:15 a . m . KEYNOTE SESSION
9:00 – 9:05 Welcome
9:05 – 9:15 Opening Remarks
9:15 – 9:20 Introductory Remarks
FSU College of Medicine Dean Alma B . Littles , M . D .
State Representative Adam Anderson ( R-Palm Harbor )
FSU President Richard D . McCullough , Ph . D .
9:20 – 10:00
KEYNOTE : Lessons Learned from 30 Years of Clinical Trials with AAV Vectors
UMass Chan Medical School Executive Deputy Chancellor and Provost Terence R . Flotte , M . D ., University of Massachusetts , Worcester
10:00 – 10:15 Discussion
President Richard D . McCullough
10:15 – 10:30 Coffee Break Sponsored by Genentech 10:30 – 12:00 p . m . SYMPOSIUM 1 : RARE DISEASE CENTERS AND PROGRAMS
10:30 – 10:35 Introduction
FSU IPRD Director Pradeep G . Bhide , Ph . D .
10:35 – 11:00
11:00 – 11:25
11:25 – 11:50
Whole genome sequencing and pharmacogenomics in a clinical setting : Lessons from the Baby Manatee Program
Genetic studies in patients with rare and undiagnosed diseases in Florida
Establishing a rare disease center : The Collaborative Center for X-Linked Dystonia Parkinsonism
David Seo , M . D ., Nicklaus Children ’ s Health System , Miami
Mustafa Tekin , M . D ., University of Miami
Nutan Sharma , M . D ., Ph . D ., Massachusetts General Hospital and Harvard Medical School , Boston
11:50 – 12:00 p . m . Discussion and Closing Remarks
IPRD Director Pradeep G . Bhide
12:00 – 1:00
Lunch Break ( on your own ) Florida Rare Disease Advisory Council ( RDAC ) meeting ( Conference Room 1160 )