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Authors
Basu, Anirban
Thomas, Simu K.
Chapman, Richard H.
Spangler, Jason
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Article History
Received: 30 May 2025
Accepted: 18 August 2025
First Online: 9 September 2025
Declarations
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: AB has engaged in private consulting work through Salutis Consulting LLC outside of the submitted work. RC is an employee of the Center for Innovation & Value Research. The Center supports its work via general funds from unrestricted membership fees. Members are listed at: . Members with interests in rare disease include Alexion AstraZeneca Rare Disease, Amgen, BIO, Boehringer Ingelheim, Bristol Myers Squibb, Dravet Syndrome Foundation, GSK, Johnson & Johnson, Eli Lilly, EveryLife Foundation for Rare Diseases, Foundation for Sarcoidosis Research, Genentech, Gilead, International Rett Syndrome Foundation, Merck, National Organization for Rare Disorders (NORD), Novartis, Otsuka, Pfizer, Takeda, and UCB. JS declares no competing interests. ST is an employee of Alexion, AstraZeneca, and holds stock or stock options in AstraZeneca.
