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Article History

Received: 28 February 2022

Accepted: 24 July 2022

First Online: 26 August 2022

Declarations

:

: The study was approved by central and local institutional review boards or ethics committees and was conducted in accordance with Good Clinical Practice guidelines and the provisions of the Declaration of Helsinki.

: Not applicable.

: Bruce Wang reports being a scientific advisor to Alnylam Pharmaceuticals and Recordati Rare Diseases. Paolo Ventura reports receiving advisory board fees and lecture fees from Alnylam Pharmaceuticals and advisory board fees from Recordati Rare Diseases. Kei-ichiro Takase reports having nothing to disclose. Manish Thapar reports being a consultant and speaker for Alnylam. David Cassiman and the University of Leuven, University Hospital Leuven report receiving research grants, travel and conference bursaries, speaker fees, and advisory board compensation from a.o. Sanofi-Genzyme, Takeda-Shire, Alexion, Alnylam, BioMarin, Actelion, Bayer, Roche, BMS, Schering-Plough, Synageva, and Chiesi. Ilja Kubisch reports receiving fees from Alnylam. Shangbin Liu reports being an employee of and owning stock and stock options in Alnylam. Marianne T. Sweetser reports being an employee of and owning stock and stock options in Alnylam. Manisha Balwani reports receiving grant support, consulting fees, advisory board fees, and lecture fees from Alnylam Pharmaceuticals, advisory board fees from Recordati Rare Diseases, grant support and advisory board fees from Mitsubishi Tanabe, and advisory board fees from Alexion, Genzyme/Sanofi, and Takeda. In addition, Mount Sinai faculty are named co-inventors with Alnylam on a patent related to the development of givosiran, the study drug. The Icahn School of Medicine at Mount Sinai receives payments related to this patent from Alnylam, and a portion of these payments are also distributed to faculty and other co-inventors.