D’Erasmo, Laura http://orcid.org/0000-0002-9174-4667
Gallo, Antonio
Cefalù, Angelo Baldassare
Di Costanzo, Alessia
Saheb, Samir
Giammanco, Antonina
Averna, Maurizio
Buonaiuto, Alessio
Iannuzzo, Gabriella
Fortunato, Giuliana
Puja, Arturo
Montalcini, Tiziana
Pavanello, Chiara
Calabresi, Laura
Vigna, Giovanni Battista
Bucci, Marco
Bonomo, Katia
Nota, Fabio
Sampietro, Tiziana
Sbrana, Francesco
Suppressa, Patrizia
Sabbà, Carlo
Fimiani, Fabio
Cesaro, Arturo
Calabrò, Paolo
Palmisano, Silvia
D’Addato, Sergio
Pisciotta, Livia
Bertolini, Stefano
Bittar, Randa
Kalmykova, Olga
Béliard, Sophie
Carrié, Alain
Arca, Marcello
Bruckert, Eric
Funding for this research was provided by:
Amryt Pharmaceutical
Article History
Received: 21 February 2021
Accepted: 24 August 2021
First Online: 8 September 2021
Declarations
:
: For the Italian cohort, informed consent was obtained from all patients before their inclusion into the study which was carried out in accordance with the ethical standards of local institutional committees for human experimentation in agreement with the Helsinki Declaration of 1964, as revised in 2018 (approval code #4928).The French cohort was declared to the ANSM (the French National Agency for Medicines safety) and received a declarant number [unique number identifying a particular research protocol, issued by the ANSM in France]: 2014-A01549-38. Two separate committees assessed the protocol of this study: French advisory committee on the processing of information for medical research (CCTIRS) and the National Commission for computer technology and freedom (CNIL) respectively in May and November 2015.Due to the retrospective nature of the present study, none of enrolled patients had received any procedures outside the standard clinical care.
: Not applicable.
: LD has received personal fees for public speaking, consultancy or grant support from Amryt Pharmaceuticals, Akcea Therapeutics, Pfizer, Amgen and Sanofi; AG has received honoraria from Amgen, Novartis, Unilever, Sanofi and Regeneron, Ackea Therapeutics, Mylan; ABC has served as a consultant for Amryt Pharmaceutical; and received lecturing fees from Amryt Pharmaceutical, MSD, Sanofi and AlfaSigma; MAV has served as a consultant for Amgen, Sanofi, Akcea, Novartin, Amgen; AP has received a lecturing fees from Sanofi and Amgen; TM has received a lecturing fees from Sanofi and Amgen; LP has received research grant support from Amgen, has served as a consultant for Akcea Therapeutics, and received lecturing fees from Sanofi, Rottapharm-MEDA; GF has received grant support from AMGEN; GI has received grant support and personal fees for public speaking from Amryt Pharmaceuticals, Akcea Therapeutics, Pfizer and Sanofi; SB has received honoraria for board, conferences, clinical trial or congress from Aegerion, Akcea, Elivie, Sanofi or Amgen; CP has received personal fees for public speaking from Amryt Pharmaceuticals; LC reports grants from Cerenis Therapeutics, grants and personal fees from MedImmune, grants from Alexion, grants from Sankyo, grants from Pfizer; MAR has received research grant support from Amryt Pharmaceutical, Amgen, IONIS, Akcea Therapeutics, Pfizer and Sanofi; has served as a consultant for Amgen, Aegerion, Akcea Therapeutics, Regeneron, Sanofi and Alfasigma and received lecturing fees from Amgen, Amryth Pharmaceutical, Pfizer, Sanofi and AlfaSigma; EB declares having received honoraria from AstraZeneca, Amgen, Genfit, MSD, Sanofi and Regeneron, Unilever, Danone, Aegerion, Chiesi, Rottapharm-MEDA, Lilly, Ionis-pharmaceuticals, Ackea Therapeutics. Other authors have declared no conflict of interest.