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Article History

Received: 25 August 2022

Accepted: 17 October 2022

First Online: 16 December 2022

Funding

: This study was sponsored by Ionis Pharmaceuticals, Inc., Carlsbad, CA, USA. Medical writing and editorial support were provided by Sandra Westra, PharmD, and Jessica D. Herr, PharmD, of Peloton Advantage, LLC, an OPEN Health company, and funded by Ionis Pharmaceuticals, Inc., Carlsbad, CA, USA. Ionis Pharmaceuticals, Inc., also provided funding for the journal’s Rapid Service Fee.

: All authors made substantial contributions to the conceptualization and design of the work and/or the acquisition, analysis, and interpretation of the data. All authors contributed to the drafting and critical review of this manuscript before providing approval for submission of the final version. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.

: Teresa Coelho: has received financial support to attend scientific meetings from Akcea, Ionis, Alnylam, Pfizer, and Biogen, and receives no personal speaker or consultant honoraria. Márcia Waddington Cruz: has received advisory board and speaker honoraria and financial support for conference attendance from Akcea, Alnylam, Pfizer, and Sobi. Chi-Chao Chao: reports no conflicts of interest. Yeşim Parman: has lectured for Alnylam and Pfizer. Jonas Wixner: has received advisory board and speaker honoraria and financial support for conference attendance from Akcea, Alnylam, and Pfizer. Markus Weiler: has received advisory board and speaker honoraria and financial support for conference attendance from Akcea, Alnylam, Pfizer, and Sobi. Fabio A. Barroso: has received advisory board and speaker honoraria and financial support for conference attendance from Alnylam, Pfizer, and PTC Therapeutics. Noel R. Dasgupta: has received advisory board honoraria from Ionis, Akcea, Alnylam, Pfizer, Eidos, and Intellia, and speaker honoraria from Akcea, Alnylam, and Pfizer. Shiangtung W. Jung: is employed by Ionis. Eugene Schneider: is employed by Ionis. Nicholas J. Viney: is employed by Ionis. P. James B. Dyck: reports no conflicts of interest. Yukio Ando: reports no conflicts of interest. Julian D. Gillmore: has served as an advisor for Alnylam, Ionis, Intellia, Eidos, Pfizer, and ATTRalus. Sami Khella: has served as a consultant to Alnylam, Ionis, Sobi, Pfizer, and Eidos. Morie A. Gertz: has received personal fees from Aptitude, Celgene, Ionis, Akcea, Janssen, Johnson & Johnson, Physicians Education Resource, Prothena, Research to Practice, and Sanofi; health grants and personal fees from Ashfield; financial support for meetings from Juno and Sorrento; fees for serving on a data safety monitoring board from AbbVie; fees for the development of educational materials from i3Health. Laura Obici: has received advisory board and speaker honoraria from Akcea, Alnylam, Pfizer, and Sobi. John L. Berk: has received honoraria for serving on advisory boards for Akcea, Ionis and Eidos, BridgeBio; scientific advisory boards for Corino, and received remotely, Intellia.

: Portions of the data in this manuscript have been presented at: the Peripheral Nerve Society, May 14–17, 2022 in Miami, Florida; the European Academy of Neurology, June 25–28, 2022 in Vienna, Austria; and the International Symposium on Amyloidosis, September 4–8, 2022.

: The protocol was approved by the institutional review board at the study sites or an independent ethics committee, and trial conduct complied with the Declaration of Helsinki of 1964 and its later amendments, and Good Clinical Practice guidelines. Written informed consent was obtained for all patients prior to enrollment.

: The datasets generated during and/or analyzed during the current study are not publicly available and the study is ongoing.