| 7:30am - 8:30am |
Breakfast and Registration |
| 8:30am - 9:00am |
Welcome Remarks/Opening
Fellowship
Announcement |
| 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 |
Noah C. Federman, MD
NTRK Fusion Cancers in Children: A model for precision oncology |
| 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 |
John Tisdale, MD
Toward Molecular Cures for the First Molecular Disease, Cell, and Gene Therapies for Sickle Cell Disease |
| 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: Breaking 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 |
