Time Agenda
8:00am - 8:30am BREAKFAST
8:30am - 9:00am WELCOME REMARKS/OPENING
9:00am - 10:00am Katherine High, MD
Turning Genes into Medicines: Highlights and Hurdles in the Development of Gene Therapy for Genetic Disease
10:00am - 10:30am John Tisdale, MD
Toward Molecular Cures for the First Molecular Disease, Cell, and Gene Therapies for Sickle Cell Disease
10:30am - 10:50am BREAK
10:50am - 11:20am Robert Nussbaum, MD
New Approaches to Variant Classification and Interpretation
11:20am - 11:50am Ellen Wright Clayton, MD, JD
Unresolved Issues about Genetic Testing and Screening in Children: Who Decides What and Why
11:50am - 1:00pm LUNCH
1:00pm - 1:30pm Noah C. Federman, MD
NTRK Fusion Cancers in Children: A model for precision oncology
1:30pm - 2:00pm Aaron Goldenberg, PhD, MA, MPH
Unresolved Issues in Gene Targeted Therapies in Pediatrics: Striving for Ethics and Equity
2:00pm - 2:30pm Nanibaa' Garrison, PhD
Unresolved Issues about Genetics and Communities: Break Barriers for Improved Access
2:30pm - 3:30pm Panel DiscussionModerator: Nanibaa' Garrison, PhD
Panelists: Katherine High, MD, John Tisdale, MD, Aaron Goldenberg, PhD, MA, MPH, Ellen Wright Clayton, MD, JD
3:30pm - 3:50pm BREAK
3:50pm - 4:20pm Lightning Round
4:20pm - 4:50pm Stanley F. Nelson, MD
Advances in Gene Therapy for Patients with Duchenne Muscular Dystrophy
4:50pm - 5:00pm Closing Remarks
5:00pm - 6:30pm Cocktails/Posters