Presentation Information
[O10-02]A phase 1/2 open-label trial of GTX102, an antisense oliogonucleotide, for patients with Angelman syndrome
○Kimimasa Tobita1,2, Tricia Cimms2, Syeda Rahman2, Angel Chen2, Wen-Hann Tan3, Kenneth Myers4, Elizabeth Berry-Kravis5, Laurent Servais6, Erick Sell7, Kimberly Goodspeed2 (1.Tokyo, Japan, 2.Ultragenyx Pharmaceutical Inc., Novato, CA, US, 3.Boston Children's Hospital, 4.Research Institute of McGill University Heath Center, Montreal, Quebec, Canada, 5.Rush University Medical Center, Chicago, US, 6.University of Oxford, Oxford, UK, 7.University of Otawa, Children's Hospital of Eastern Ontario)