Gentile, Luca http://orcid.org/0000-0001-7947-8610
Mazzeo, Anna http://orcid.org/0000-0003-1914-6184
Briani, Chiara http://orcid.org/0000-0001-8035-0200
Casagrande, Silvia http://orcid.org/0000-0002-4111-1810
De Luca, Marcella http://orcid.org/0009-0006-4913-9232
Fabrizi, Gian Maria http://orcid.org/0000-0001-6804-0226
Gagliardi, Christian http://orcid.org/0000-0001-7432-8023
Gemelli, Chiara http://orcid.org/0000-0003-1911-2726
Forcina, Francesca http://orcid.org/0009-0002-2572-5801
Grandis, Marina http://orcid.org/0000-0001-6637-4425
Guglielmino, Valeria http://orcid.org/0000-0002-9049-5988
Iabichella, Giacomo http://orcid.org/0009-0000-9256-0184
Leonardi, Luca http://orcid.org/0000-0002-1267-864X
Lozza, Alessandro http://orcid.org/0000-0001-5622-2647
Manganelli, Fiore http://orcid.org/0000-0002-1442-9604
Mussinelli, Roberta http://orcid.org/0000-0003-2950-3413
My, Filomena
Occhipinti, Giuseppe http://orcid.org/0009-0005-8061-7645
Fenu, Silvia http://orcid.org/0000-0002-5233-6580
Russo, Massimo http://orcid.org/0000-0002-6017-370X
Romano, Angela http://orcid.org/0000-0002-2061-1509
Salvalaggio, Alessandro http://orcid.org/0000-0002-1273-7566
Tagliapietra, Matteo http://orcid.org/0000-0002-3048-1453
Tozza, Stefano http://orcid.org/0000-0002-9672-4577
Palladini, Giovanni http://orcid.org/0000-0001-5994-5138
Obici, Laura http://orcid.org/0000-0001-7468-700X
Luigetti, Marco http://orcid.org/0000-0001-7539-505X
Article History
Received: 27 October 2023
Accepted: 23 March 2024
First Online: 16 April 2024
Declarations
:
: Dr Luigetti received financial grants (honoraria and speaking) from Ackea, Alnylam, Sobi, and Pfizer, and travel grants from Ackea, Alnylam, Sobi, Pfizer, Kedrion and Grifols.Dr. Gentile received financial grants (honoraria and speaking) from Akcea, Alnylam, and Pfizer, and travel grants from Alnylam and CSL Behring.Dr. Mazzeo received speaker fees and consulting honoraria from Alnylam Pharmaceuticals, Akcea Therapeutics, and Pfizer Inc.Dr. Manganelli has no conflict of interest to declare.Dr Obici reports speaker and consulting honoraria from Pfizer, Alnylam, SOBI, Novo Nordisk, Astra Zeneca and BridgeBio.Dr. Briani received speaker and consulting honoraria from Alnylam, Ionis, and Pfizer, and travel grants from Kedrion, Alnylam and CSL Behring.Dr. Casagrande has no conflict of interest to declare.Dr. De Luca received financial grants (speaking) from Pfizer, and travel grants to attend scientific meetings from Pfizer and SOBI.Dr. Fabrizi received financial grants (honoraria and speaking) from Akcea, Alnylam and travel grants from Akcea, Pfizer and Kedrion.Dr. Gagliardi received financial grants (consulting and speaking) from Pfizer and Alnylam.Dr. Gemelli received financial grants (speaking) from Italfarmaco, and travel grants from Biogen for participation in national meetings.Dr. Forcina has no conflict of interest to declare.Dr. Grandis acknowledges donations from Sanofi Genzyme to support research activities of her Research Unit. Financial support from Pfizer, Alnylam and Sanofi Genzyme for participation in National and International Meetings. Participation in Advisory Board of Pfizer and Argenx. Speaker honorarium from Sanofi Genzyme and Alexion.Dr. Guglielmino has no conflict of interest to declare.Dr. Iabichella received travel grants to attend scientific meetings from Alnylam and SOBI.Dr. Leonardi received financial grants (honoraria and speaking) from Alnylam, and travel grants to attend scientific meetings from Akcea, SOBI, Alnylam.Dr. Lozza has no conflict of interest to declare.Dr. Mussinelli received financial grants (speaking) from Pfizer and SOBI, and travel grants to attend scientific meetings from Alnylam.Dr. My received financial grants (honoraria and speaking) from Alnylam, and travel grants to attend scientific meetings from Alnylam.Dr. Occhipinti has no conflict of interest to declare.Dr. Fenu acknowledges financial support from Alnylam and Pfizer for participation in national and international meetings.Dr. Russo has no conflict of interest to declare.Dr. Romano received financial grants (honoraria and speaking) from Akcea, and travel grants to attend scientific meetings from Akcea, Alnylam, Pfizer, and Csl Behring.Dr. Salvalaggio received travel grant to attend scientific meeting from Alnylam.Dr. Stefano Tozza received personal fees for scientific events from Alnylam Pharmaceuticals, Amicus Therapeutics and Takeda Pharmaceutical Co, travel grants to attend scientific meetings from Akcea Therapeutics.
: The study protocol was approved by Ethics Committee of Unit of Neurology and Neuromuscular Diseases, AOU G. Martino, Messina, i.e., the coordinating centre (protocol number: 109-21) and subsequently by the Ethics Committees of the participating centres.
: Participants provided informed consent.