9AM - 10:20AM Session I — Just for Parents: Research, Studies & Data Collection
As we move ever closer to bringing effective treatments to our children, we need our community caregivers’ assistance to ensure we are designing the best possible clinical trials and outcome measures.
This entails everything from participating in online surveys to enrolling in clinical research studies.
Every parent plays a critical role in making sure we are successful. Listen to speakers from Boston Children’s Hospital, Roche, RTI International, the Angelman Biomarkers and Outcome Measures Alliance and more.
They will explain how you can help move us forward in our mission to cure Angelman syndrome.
9AM - 9:20AM Terry Jo Bichell, Ph.D., director, Angelman Biomarkers and Outcome Measures Alliance
9:20AM - 9:40AM Meagan Cross, chairwoman, FAST Australia
9:40AM - 10AM Anjali Sadhwani, Ph.D., clinical psychologist, Boston Children's Hospital
Anne Wheeler, Ph.D., public health researcher, RTI International
10AM - 10:20AM Meghan Miller, Ph.D., discovery project leader, Roche
10:20AM - 10:30AM Break
10:30AM - 12:30PM Session II — In the Clinic
Exciting clinical trial updates from Ovid Therapeutics and Disruptive Nutrition
10:30AM - 11:30AM Jeremy Levin, D.Phil., MB BChir, chairman and chief executive officer, Ovid Therapeutics Inc.
Amit Rakhit, M.D., MBA, chief medical and portfolio officer, Ovid Therapeutics Inc.
11:30AM - 12:30PM Mike McCandless, founder, Disruptive Nutrition Donna Herber, Ph.D., chief science officer, Disruptive Nutrition Jessica Duis, assistant professor of pediatrics, Vanderbilt University Medical Center
12:30PM - 1:30PM Complimentary Lunch for Summit Attendees
1:30PM - 4:15PM Session III — Towards a Cure: Gene Therapy for the Treatment of AS
Currently, there are multiple gene therapy strategies in development to treat Angelman syndrome.
These strategies will be discussed in detail.
1:30PM - 1:50PM FAST Research Agenda Overview
Allyson Berent, D.V.M., chief science officer, FAST
2PM - 3PM Keynote: James M. Wilson, M.D., Ph.D., Rose H. Weiss professor and director, Orphan Disease Center; director, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania
3PM - 3:15PM Break
3:15PM - 4:15PM Jodi Cook, Ph.D., chief operating officer, Agilis Biotherapeutics
Edwin Weeber, Ph.D., professor, University of South Florida
Jennifer Daily, Ph.D., director of research, Agilis Biotherapeutics
4:30PM - 5:30PM Session IV— Gene Therapy Round Table: Ask the Experts
Not sure what a viral vector is? Can’t pronounce antisense oligonucleotide? Not sure how treating a genetic disorder is even possible? Come ask the experts! Dr. Jim Wilson, University of Pennsylvania; Dr. Arthur Beaudet, Baylor College of Medicine; Dr. Scott Dindot, Texas A&M University; Dr. Kevin Nash, University of South Florida; Dr. Edwin Weeber, University of South Florida; and Dr. David Segal, University of California, Davis, will be taking questions from the audience

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