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Article History

Received: 21 January 2025

Accepted: 1 April 2025

First Online: 11 April 2025

Declarations

:

: The study was conducted in accordance with consensus ethics principles derived from international ethics guidelines, including the Declaration of Helsinki and Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, the International Conference on Harmonization (ICH) Good Clinical Practices (GCP) Guideline [E6], and applicable laws and regulations. The protocol and consent forms were approved by local institutional review boards at each study site. Written informed consent was obtained from at least 1 parent or guardian, and written assent was obtained from patients per local regulations, before any study procedures were performed. All animals were handled in compliance with National Institutes of Health and institutional guidelines that were approved by the institutional animal care and use committees of the University of Pennsylvania and University of Florida, as previously documented (Hammers et al. 2016).

: Not applicable.

: DC is a full-time employee and shareholder of Eli Lilly and Company. At the time the study was performed and this manuscript was initiated, JL (retired) was a full-time employee of Eli Lilly Canada. BJB has received research support from Sarepta Therapeutics, Pfizer and is a member of the Global Pompe Advisory Board supported by Sanofi. BJB has received consulting fees from Amicus Therapeutics, Rocket Pharma, and Tenaya. BJB is a co-founder of Ventura Life Sciences. BJB is an uncompensated member of the MDA Board or Directors. DWH and HLS declared no competing interests.