Document is current

Article History

Received: 19 January 2018

Accepted: 24 September 2018

First Online: 29 October 2018

Ethics approval and consent to participate

: The study complied with the Declaration of Helsinki. For the treatment period from 1993 to August 2007, the study was approved by the Ethics Committee of Bicêtre Hospital (Comité de Protection des Personnes, Hôpital Bicêtre, Le Kremlin-Bicêtre, France) and written informed consent was obtained from parents prior to enrollment, as previously reported []. Since August 2007 to September 2013, patients benefited from a “Autorisation Temporaire d’Utilisation” delivered for each patient by the “Agence Nationale de Sécurité du Médicament et des Produits de Santé (France)”. Thereafter, the European Commission granted a marketing authorisation valid throughout the European Union for Orphacol on September 2013 []. Therefore, for the 10 year period from August 2007 to 2017 which represents the basis of the study, consent to participate was not needed.

: Not applicable.

: EJ consulted for laboratory CTRS (France). EG received support for travel from CTRS, Alexion, Spindler Mayoli, Albireo. The other authors reported no conflict of interest.

: Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.