11
Participants
Start Date
October 31, 2019
Primary Completion Date
January 1, 2021
Study Completion Date
January 1, 2021
Deflazacort
Deflazacort tablet will be administered as per the dose and schedule specified in the arm.
Rigshospitalet, University of Copenhagen, Copenhagen
University Hospital La Timone, Marseille
University of Pennsylvania, Philadelphia
Hugo W Moser Research Institute at Kennedy Krieger Institute, Baltimore
Rare Disease Research, LLC, Atlanta
Sahlgrenska University Hospital, Gothenburg
University of Iowa Hospitals and Clinics, Iowa City
CHRU de NANCY Service de Neurologie, France
University of Minnesota, Minneapolis
Washington University School of Medicine, St Louis
The University of Kansas Medical Center, Kansas City
Ludwig-Maximilians University Munich, Friedrich-Baur-Institute, Munich
University of Washington, Seattle
Pirogov Russian National Research Medical University, Moscow
Saint-Petersburg State Pediatric Medical University, Saint Petersburg
University of Alberta, Edmonton
Ottawa Hospital, Ottawa
Oslo University Hospital, Oslo
Lead Sponsor
PTC Therapeutics
INDUSTRY