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7:30am - 8:30am
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BREAKFAST AND REGISTRATION
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8:30am - 9:00am
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WELCOME REMARKS/OPENING
Fellowship Announcement
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9:00am - 10:00am
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Katherine High, MD
Turning Genes into Medicines: Highlights and Hurdles in the Development of Gene Therapy for Genetic Disease
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10:00am - 10:30am
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Noah C. Federman, MD
NTRK Fusion Cancers in Children: A model for precision oncology
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10:30am - 10:50am
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BREAK
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10:50am - 11:20am
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Robert Nussbaum, MD
New Approaches to Variant Classification and Interpretation
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11:20am - 11:50am
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Ellen Wright Clayton, MD, JD
Unresolved Issues about Genetic Testing and Screening in Children: Who Decides What and Why
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11:50am - 1:00pm
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LUNCH
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1:00pm - 1:30pm
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John Tisdale, MD
Toward Molecular Cures for the First Molecular Disease, Cell, and Gene Therapies for Sickle Cell Disease
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1:30pm - 2:00pm
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Aaron Goldenberg, PhD, MA, MPH
Unresolved Issues in Gene Targeted Therapies in Pediatrics: Striving for Ethics and Equity
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2:00pm - 2:30pm
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Nanibaa' Garrison, PhD
Unresolved Issues about Genetics and Communities: Breaking Barriers for Improved Access
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2:30pm - 3:30pm
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Panel Discussion
Moderator: Nanibaa' Garrison, PhD
Panelists: Katherine High, MD, John Tisdale, MD, Aaron Goldenberg, PhD, MA, MPH, Ellen Wright Clayton, MD, JD
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3:30pm - 3:50pm
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BREAK
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3:50pm - 4:20pm
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Lightning Round
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4:20pm - 4:50pm
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Stanley F. Nelson, MD
Advances in Gene Therapy for Patients with Duchenne Muscular Dystrophy
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4:50pm - 5:00pm
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Closing Remarks
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5:00pm - 6:30pm
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Cocktails/Posters
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