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Authors
Nandi, Pooja
Ellis, Robert https://orcid.org/0000-0002-9772-7446
Hiros, Jennifer
Howard, Paul
Veleva-Rotse, Biliana O.
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Funding
Funding for this research was provided by:
Amicus Therapeutics, Inc. (Amicus Therapeutics, Inc.)
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Article History
Received: 20 December 2024
Accepted: 30 June 2025
First Online: 28 July 2025
Declarations
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: Ethical approval was not required for this study as it involved an analysis of publicly available data and published literature and advisory board discussions; patients and clinician advisors gave their consent to participate in the advisory boards and for the information they provided to be published.
: In addition to what is noted in the ‘Ethics approval and consent to participate’ section above, all individuals named in the Acknowledgments section have provided written consent for publication.
: Pooja Nandi was an employee of Koneksa Health and owned company stock at the time this work was conducted. Robert Ellis is an employee of Koneksa Health and owns company stock. Biliana O. Veleva-Rotse, Jennifer Hiros, and Paul Howard are employees and stockholders of Amicus Therapeutics, Inc.
