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Achieving clinical trial readiness in Angelman syndrome — a model for other neurogenetic disorders

April 11, 2023 from 10:00 am to 10:50 am

Chairs: Anjali Sadhwani, Boston Children’s Hospital & Anne Wheeler, RTI International, University of North Carolina at Chapel Hill

Discussant: Dominique Pichard, International Rett Syndrome Foundation


  • From bench to bedside – The evolution of treatment options for Angelman syndrome: Elizabeth Jalazo, University of North Carolina at Chapel Hill
  • The importance of natural history studies and linking data from multiple sources for the development of interventions and treatments in Angelman syndrome: Katherine Andersone, Boston Children’s Hospital, Department of Psychiatry and Behavioral Sciences, Boston, MA; Sarah Nelson Potter, RTI International; Angela Gwaltney, RTI International; Sonya Powers, ; Anne Wheeler, RTI InternationalAmanda Moore, Angelman syndrome Foundation  Anjali Sadhwani, Boston Children’s Hospital  Lynne Bird, UCSF  Wen-Hann Tan, Boston Children’s Hospital
  • Outcome measures and clinical endpoints for Angelman syndrome – What is meaningful?: Angela  Gwaltney, RTI International; Sonya  Powers, ; Sarah Nelson Potter, RTI International; Katherine Anderson, Boston Children’s Hospital; Anne  Wheeler, RTI InternationalAnjali Sadhwani, Boston Children’s Hospital