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Article History

Received: 30 March 2023

Accepted: 25 October 2023

First Online: 9 November 2023

Declarations

:

: Each subject was required to sign an informed consent form (ICF) to participate in the study. A legally authorized representative could have consented on behalf of a subject who was otherwise unable to provide informed consent, if acceptable to and approved by the site and/or the site’s institutional review board (IRB)/independent ethics committee (IEC). An ICF that explained the procedures of the study, including the potential hazards, was reviewed and approved by the IRB/IEC before its use. The ICF was read by and explained to each subject or their legally authorized representative before entering the study. Each subject had ample opportunity to ask questions and was assured of the right to withdraw from the study at any time without any disadvantage and without having to provide a reason for this decision. Each subject or representative received a signed and dated copy of the ICF. The study was conducted in accordance with the Declaration of Helsinki.

: Not applicable.

: DAA is a former employee of Endpoint Outcomes and a current employee of BioMarin Pharmaceutical, Inc. RFG received research funding from Agios Pharmaceuticals Inc., Novartis, and Sobi and consultancy from Agios Pharmaceuticals Inc., and Sanofi. RJK reports consultancy from Agios Pharmaceuticals Inc., Amgen, Biogen Canada Limited, Hoffman‐LaRoche Ltd, Novo Nordisk Canada Inc, Octapharma AG, Sanofi Genzyme, and Takeda Canada Inc. SS reports consultancy for Merck Healthcare KGaA and education grants from GlaxoSmithKline and The European Haematology Association. JL, FT, and ANB are employees and shareholders of Agios Pharmaceuticals Inc. EZ is a former employee and shareholder of Agios Pharmaceuticals Inc. AJ and BF are employees of Endpoint Outcomes (now Lumanity).