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Article History

Received: 10 April 2023

Accepted: 5 September 2023

First Online: 12 October 2023

Declarations

:

: Not applicable.

: Not applicable.

: YA reports financial support for the submitted work from Pfizer; payment or honoraria from Pfizer, Alnylam Pharmaceuticals, and Prothena; and payment for expert testimony from Pfizer, Alnylam Pharmaceuticals, and Prothena outside the submitted work. MWC reports financial support for the submitted work from Pfizer and consulting fees from Pfizer outside the submitted work. YS reports financial support for the submitted work from Pfizer and grants, consulting fees, and payment or honoraria from Pfizer and Alnylam Pharmaceuticals outside the submitted work. H Koike reports financial support for the submitted work from Pfizer and consulting fees, payment or honoraria, and support for meetings and travels from Pfizer and Alnylam Pharmaceuticals outside the submitted work. MU reports financial support for the submitted work from Pfizer and grants, payment or honoraria, and support for meetings and travels from Pfizer and Alnylam Pharmaceuticals outside the submitted work. H Konishi and TI are full-time employees of Pfizer Japan Inc. TC reports financial support for the submitted work from Pfizer and grants from the THAOS Registry; payment or honoraria from Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Akcea Therapeutics, Sobi, Biogen, and Roche; and support for meetings and travels from Pfizer, Alnylam Pharmaceuticals, Sobi, and Biogen outside the submitted work. TC also declares participation in the data safety monitoring or advisory boards for Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Akcea Therapeutics, Sobi, Biogen, and Roche and serves as head of the Portuguese Society for the Study of Neuromuscular Disorders.