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Article History

Received: 30 September 2025

Accepted: 30 October 2025

First Online: 28 November 2025

Change Date: 10 January 2026

Change Type: Update

Change Details: The original article was revised due to update in Tale 2

Declarations

:

: Julie E. Hoover-Fong has received grants or contracts from clinical trials from BioMarin, BridgeBio Pharma and Pfizer, has received consulting fees from BioMarin, BridgeBio Pharma, NovoNordisk and Innoskel, has received honoraria from Medscape and BioMarin, and has participated on advisory boards with MCDS-Therapy Clinical Trial (unpaid), BioMarin and Bridgebio Pharma. Ravi Savarirayan has received consulting fees and grants from BioMarin, has participated on advisory boards with Ascendis and BioMarin, and has received consulting fees from BridgeBio Pharma. Anne Grumet, Daniela Rogoff, and Elena Muslimova,are employees and shareholders of BridgeBio Pharma. Susan D. Mathias is an employee and Hilary H. Colwell is a consultant to Health Outcomes Solutions, which received funding from BridgeBio Pharma for undertaking this research. Inês Alves, Chandler Crews, Amer Haider, and Susana Noval Iruretagoyena report no competing interests.

: Human participants’ research approval for the study was provided by an independent scientific review committee, WCG IRB (20214002). All participants that contributed data provided informed consent as part of the research associated with data collection. This study was performed in accordance with the Helsinki Declaration of 1964 and its later amendments.