4th Annual Family Weekend

Elliott Sherr MD PhD: An Update of DDX3X Findings to date

Professor of Neurology, Pediatrics
Institute of Human Genetics
UC San Francisco

Maria L Escolar, MD. MS.: The Importance of Natural History Studies

Associate Professor of Pediatrics
University of Pittsburgh
Director, Program for the Study of Neurodevelopment in Rare Disorders
Children’s Hospital of Pittsburgh of UPMC

Debra L. Silver, Ph.D. How DDX3X controls brain development

Associate Professor, Departments of Molecular Genetics and Microbiology, Cell Biology, and Neurobiology
Director of Graduate Studies, Duke Developmental and Stem Cell Biology (DSCB) Program
Investigator, Duke Institute for Brain Sciences

Randy J. Chandler, PhD, MB: An overview of gene therapy for neurological disorders

Staff Scientist
Genetic and Molecular Biology Branch
National Human Genome Research Institute
National Institutes of Health

Kevin Francis PhD: Importance of Stem Cell therapies in Rare Diseases

Assistant Scientist – Cellular Therapies and Stem Cell Biology Group, Sanford Research
Assistant Professor – Department of Pediatrics, Sanford School of Medicine, University of South Dakota

Karlla W. Brigatti, MS LCGC: Why scientific research is so important

Genetic Counselor & Research Operations Director
Clinic for Special Children

Rick Guidotti, Photographer: Seeing beauty in all differences

positiveexposure.org
And he will photograph all the kids after his talk!