255
Participants
Start Date
July 31, 2024
Primary Completion Date
February 28, 2029
Study Completion Date
February 28, 2029
GTX-102
Antisense Oligonucleotide
The Royal Children's Hospital, Melbourne
Queensland Children's Hospital, South Brisbane
Weill Cornell Medicine, New York
AP-HM - Hopital de la Timone, Marseille
Universitaetsklinikum Hamburg-Eppendorf, Hamburg
Hospital Universitario Puerta de Hierro, Majadahonda
Rare Disease Research, LLC, Atlanta
Rush University Medical Center, Chicago
AP-HP Hopital Necker-Enfants Malades, Paris
University of California, Los Angeles (UCLA), Los Angeles
University of California, San Diego - Rady Children's Hospital, San Diego
Sheba Medical Center, Ramat Gan
Boston Children's Hospital, Boston
MAGIC Clinic Ltd, Calgary
British Columbia Children's Hospital, Vancouver
Childrens Hospital London Health Sciences Centre, London
Childrens Hospital of Eastern Ontario, Ottawa
The Research Institute of the McGill University Health Centre, Montreal
Universitaetsklinikum Leipzig, Leipzig
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell, Sabadell
University of Cambridge, Cambridge
Great Ormond Street Hospital for Children, London
Oxford University Hospitals NHS Foundation Trust, Oxford
Ultragenyx Pharmaceutical Inc
INDUSTRY