Funding for this research was provided by:
Fondation Saint Luc (mandat de recherche temps plein)
Fonds pour la Formation à la Recherche dans l’Industrie et dans l’Agriculture (F 3/5/5 – FRIA/FC)
Received: 27 October 2019
Revised: 17 December 2019
Accepted: 26 January 2020
First Online: 7 February 2020
Ethics approval and consent to participate
: Ethical approval to report this case series was obtained from Belgian regulatory authorities and the ethical committee (2011/04OCT/388). All procedures in this study were conducted in accordance with the European Medicines Agency’s (EMA) pediatric committee (EudraCT number: 2011-004074-28) and conducted according to the 2000 revised principles of the Declaration of Helsinki (ClinicalTrials.gov identifier: NCT01765283). Written informed consent was obtained from a legally authorized representative(s) for anonymized patient information to be published in this article.
: Written informed consent was obtained from a legally authorized representative(s) for anonymized patient information to participate in this study.
: E.S is a founder and scientific advisor for the Promethera Biosciences and has founding shares and/or stock options. F.S. is the principal Investigator for the Promethera Biosciences. X.S. has a consultancy agreement with the Promethera Biosciences. This study was supported in part by research funding from the Promethera Biosciences. Other authors declare that they have no competing financial interests. All authors declare to have no non-financial conflict of interests.