Bertholet-Thomas, Aurélia
Claramunt-Taberner, Debora
Gaillard, Ségolène
Deschênes, Georges
Sornay-Rendu, Elisabeth
Szulc, Pawel
Cohen-Solal, Martine
Pelletier, Solenne
Carlier, Marie-Christine
Cochat, Pierre
Bacchetta, Justine
Funding for this research was provided by:
Raptor Pharmaceuticals
Article History
Received: 19 September 2017
Revised: 8 January 2018
Accepted: 23 January 2018
First Online: 12 February 2018
Compliance with ethical standards
: The study was approved by the local institutional review board (Comité de Protection des Personnes Lyon Sud Est II, initial approval of the Crystobs study 9/8/2010, approval of the 5th amendment 9/7/2016). Written informed consent was obtained from all patients (and parents in case of pediatric patients).
: JB has received a travel grant from Raptor Pharmaceuticals and an educational research grant for this clinical study.PC is a medical expert for Raptor Pharmaceuticals and co-investigator for the RP 103-3, 103-4, and 103-7 studies.ABT is a medical expert for Raptor Pharmaceuticals and co-investigator for the RP 103-3, 103-4, and 103-7 studies.MCS received travel grants from Amgen.