Updates are available Correction dated 2025-07-31
Click to view Correction: https://doi.org/10.1007/s00415-025-13147-4
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Authors
Berger, Kenneth I. https://orcid.org/0000-0003-4879-6071
Chien, Yin-Hsiu
Dubrovsky, Alberto
Kishnani, Priya S.
Llerena, Juan C. Jr.
Neilan, Edward
Roberts, Mark
Sheng, Bun
Batista, Julie L.
Periquet, Magali
Wilson, Kathryn M.
van der Ploeg, Ans T.
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Clinical Trials BETA
Clinical trials referenced in this document:
nct00231400 at ClinicalTrials.govDocuments that mention this clinical trial
Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry
https://doi.org/10.1007/s00415-024-12489-9
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Funding
Funding for this research was provided by:
Sanofi
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More Information
Article History
Received: 22 February 2024
Revised: 28 May 2024
Accepted: 29 May 2024
First Online: 19 June 2024
Change Date: 31 July 2025
Change Type: Correction
Change Details: A Correction to this paper has been published:
Change Details: https://doi.org/10.1007/s00415-025-13147-4
Declarations
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: KIB has served on advisory boards for AskBio, Sanofi, Spark Therapeutics, and Takeda and has received consulting fees from Amicus Therapeutics, AskBio, Inventiva Pharma, Sanofi, Spark Therapeutics, and Takeda during the conduct of the study. He is employed by Sanofi at the time of manuscript submission. Y-HC has served on advisory boards for Amicus and Sanofi; has received consulting fees from Amicus and Sanofi; has undertaken contracted research for Sanofi; and received honoraria from Biogen, Novartis, Sanofi, and Takeda. AD has served on advisory boards for Sanofi. PSK has served on advisory boards for Amicus Therapeutics, Baebies, and Sanofi; has received consulting fees from Amicus Therapeutics, Asklepios Biopharmaceutical, Inc. (AskBio), JCR Pharma, Maze Therapeutics, and Sanofi; has undertaken contracted research for Amicus Therapeutics and Sanofi; and has ownership interest < 5% (stocks, stock options or other ownership interest excluding diversified mutual funds) in AskBio and Maze Therapeutics; and has received travel expenses from Amicus Therapeutics and Sanofi. JL has no competing interests to declare that are relevant to the content of this article. EN was employed by Sanofi and held shares of stock in the company at the time of the study. He is currently employed by the National Organization for Rare Disorders. MR has served on advisory boards for Sanofi (Pompe Registry); has received consulting fees from Amicus Therapeutics, BioMarin, and Sanofi, and has undertaken contracted research for Amicus Therapeutics. BS has served on advisory boards for Sanofi (Pompe Registry). JLB is a Sanofi employee and may hold shares and/or stock options in the company. MP was an employee of Sanofi at the time of study participation and may hold shares and/or stock options in the company; she is currently employed by UCB. KW is a contractor for Navitas Data Sciences and is funded by Sanofi. AvdP has served on advisory boards for Amicus, BioMarin, Sanofi, and Spark Therapeutics; has received consulting fees from Amicus, BioMarin, Sanofi, and Spark Therapeutics; and has undertaken contracted research for Amicus, BioMarin, Sanofi, and Spark Therapeutics.
: Local sites’ Institutional Review Boards or Independent Ethics Committees approved the Pompe Registry protocol, informed consent form, and local authorization documents for data submissions. Each participating center obtained written informed consent from patients or for minors, their guardians, to submit their data to the Pompe Registry and to use and disclose this information anonymously in aggregate analyses.
