Brannagan, Thomas H.
Coelho, Teresa
Wang, Annabel K.
Polydefkis, Michael J.
Dyck, Peter J.
Berk, John L.
Drachman, Brian
Gorevic, Peter
Whelan, Carol
Conceição, Isabel
Plante-Bordeneuve, Violaine
Merlini, Giampaolo
Obici, Laura
Plana, Josep Maria Campistol
Gamez, Josep
Kristen, Arnt V.
Mazzeo, Anna
Gentile, Luca
Narayana, Arvind
Olugemo, Kemi
Aquino, Peter
Benson, Merrill D.
Gertz, Morie
,
Funding for this research was provided by:
Akcea Therapeutics
Article History
Received: 16 March 2022
Revised: 7 July 2022
Accepted: 8 July 2022
First Online: 31 July 2022
Declarations
:
: TB reports being an advisory board member for Akcea Therapeutics, Pfizer, and Alnylam Pharmaceuticals; study investigator for Ionis Pharmaceuticals, Inc., and Alnylam Pharmaceuticals; and speaker for Alnylam Pharmaceuticals; and receiving honoraria for speaking for Akcea Therapeutics. TC reports receiving financial support to attend scientific meetings from Pfizer, Alnylam, Ionis, Akcea, and Biogen. AKW reports being study investigator, consultant, and speaker for Ionis Pharmaceuticals, Inc.; consultant for Pfizer; and study investigator for Alnylam. MJP reports receiving honoraria from Pfizer and Alnylam Pharmaceuticals, Inc. PJD reports receiving training and consulting honoraria from Ionis Pharmaceuticals, Inc., and Alnylam Pharmaceuticals. JLB reports receiving honoraria from Ionis Pharmaceuticals, Inc., and Alnylam Pharmaceuticals; and being a study investigator for Ionis Pharmaceuticals, Inc., Alnylam Pharmaceuticals, Eidos BridgeBio, and Pfizer. BD reports being a consultant/advisory board member for Eidos and Alnylam Pharmaceuticals. PG declares no competing interests. CW reports being an advisory board member for Alnylam and Akcea Therapeutics. IC reports being a consultant for Alnylam and Pfizer; receiving honoraria for Alnylam, Pfizer, and Sanofi; and being on speaker bureaus for Alnylam, Pfizer, and Sanofi. VP-B reports receiving consulting honoraria for Alnylam Pharmaceuticals, Ionis, and Pfizer and travel honoraria from Ionis. GM declares no competing interests. LO reports receiving speaker honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals, SOBI, and Pfizer. JMCP declares no competing interests. JG declares no competing interests. AVK reports receiving honoraria and fees for lectures and speakers bureaus from Alnylam Pharmaceuticals, Akcea Therapeutics, and Pfizer Inc. AM reports receiving speaker fees and consulting honoraria from Alnylam Pharmaceuticals, Akcea Therapeutics, and Pfizer Inc. LG reports receiving speaker fees and consulting honoraria from Pfizer. AN is a former employee of Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals, Inc. KO is a former employee of Ionis Pharmaceuticals, Inc. PA is a former employee of Ionis Pharmaceuticals, Inc./Akcea Therapeutics. MDB reports being study investigator for Ionis Pharmaceuticals, Inc. MG reports receiving consulting honoraria from Ionis, AbbVie, Alnylam, Amgen, Annexon, Appellis, Celgene, Janssen, Medscape, Physicians Education Resource, Prothena, Research to Practice, Teva, and Spectrum, and grants from Spectrum.
: The trial protocol for the OLE study was approved by the relevant institutional review boards or local ethics committees and regulatory authorities. The trial was conducted in accordance with Good Clinical Practice guidelines of the International Conference on Harmonisation and the principles of the Declaration of Helsinki. All patients provided written informed consent to participate in the study.