Funding for this research was provided by:
PTC Therapeutics (NA)
Text and Data Mining valid from 2020-01-09
Received: 20 August 2019
Accepted: 30 December 2019
First Online: 9 January 2020
Ethics approval and consent to participate
: Not applicable; no patient-specific identifier or personal information is presented in this report.
: Written informed consent was obtained from the parents of all individual participants included in the case report.
: Berrin Monteleone, MD: Consultant to Agilis Biotherapeutics/PTC Therapeutics, GTC, Inc.Keith Hyland, PhD: Consultant for Censa Pharmaceuticals and Agilis Biotherapeutics/PTC Therapeutics, GTC, Inc.