Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.

"Declarations Ethics approval and consent to participateLegal guardians of all patients provided informed written consent to submit their health information to the Registry, to be used for further analysis and data sharing; independent registry sites are responsible for ensuring this compliance. The Registry protocol, informed consent form, and any locally required authorization documents are reviewed and approved by the local fully constituted Institutional Review Board or Independent Ethics Committee. Consent for publicationNot applicable. Competing interestsPriya S. Kishnani: Advisory Board: Amicus, Baebies, Sanofi; Consulting Fees: Amicus, AskBio, Sanofi, Vertex, Maze therapeutics. Contracted Research: Amicus, Sanofi, Valerion. Honoraria: Amicus, AskBio, Sanofi, Vertex. Ownership Interest less than 5% (stocks, stock options or other ownership interest excluding diversified mutual funds): AskBio, Baebies. Travel Expenses: Amicus, Sanofi. David Kronn: Advisory Board: AskBio and Sanofi; Contracted research: Sanofi; Speaker’s bureau: Sanofi; Travel expenses: AskBio and Sanofi. Shugo Suwazono: Advisory board: Sanofi. Alexander Broomfield: Advisory board: Sanofi and Honoraria: Orchard Therapeutics and Sanofi. Juan Llerena: Advisory Board: Sanofi, Biomarin; Consulting Fees: Sanofi, Biomarin; Travel expenses: Sanofi, Biomarin. Zuhair Nasser Al-Hassnan: Advisory board: Sanofi. Andreas Hahn: Advisory board: Sanofi and Amicus Therapeutics; Honoraria and consulting fees: Sanofi, Amicus, Takeda, Avrobio, and Biomarin. Yin-Hsiu Chien: Advisory board: Amicus Therapeutics and Sanofi, Contracted research: Sanofi, Honoraria, and consulting fees from Sanofi. Kathryn M. Wilson: contractor for Navitas Data Sciences, funded by Sanofi. Julie L. Batista, Nadia Daba, and Magali Periquet: employees of Sanofi may hold shares and stock options. Competing interests Priya S. Kishnani: Advisory Board: Amicus, Baebies, Sanofi; Consulting Fees: Amicus, AskBio, Sanofi, Vertex, Maze therapeutics. Contracted Research: Amicus, Sanofi, Valerion. Honoraria: Amicus, AskBio, Sanofi, Vertex. Ownership Interest less than 5% (stocks, stock options or other ownership interest excluding diversified mutual funds): AskBio, Baebies. Travel Expenses: Amicus, Sanofi. David Kronn: Advisory Board: AskBio and Sanofi; Contracted research: Sanofi; Speaker’s bureau: Sanofi; Travel expenses: AskBio and Sanofi. Shugo Suwazono: Advisory board: Sanofi. Alexander Broomfield: Advisory board: Sanofi and Honoraria: Orchard Therapeutics and Sanofi. Juan Llerena: Advisory Board: Sanofi, Biomarin; Consulting Fees: Sanofi, Biomarin; Travel expenses: Sanofi, Biomarin. Zuhair Nasser Al-Hassnan: Advisory board: Sanofi. Andreas Hahn: Advisory board: Sanofi and Amicus Therapeutics; Honoraria and consulting fees: Sanofi, Amicus, Takeda, Avrobio, and Biomarin. Yin-Hsiu Chien: Advisory board: Amicus Therapeutics and Sanofi, Contracted research: Sanofi, Honoraria, and consulting fees from Sanofi. Kathryn M. Wilson: contractor for Navitas Data Sciences, funded by Sanofi. Julie L. Batista, Nadia Daba, and Magali Periquet: employees of Sanofi may hold shares and stock options."

"Funding The Pompe Registry is funded by Sanofi."