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Article History

Received: 16 November 2025

Accepted: 27 February 2026

First Online: 24 March 2026

Conflict of Interest

: Melody S. Benton is principal investigator and lead researcher for the study; the project is sponsored/funded by BioMarin. Dongzhe Hong declares that he has no competing interests. Mark W. Skinner’s institution has received independent research funding from Bayer, BioMarin, Chugai, CSL Behring, Roche, Sanofi, Spark, and Takeda. His institution has received fees or honoraria from Bayer, BioMarin, Genentech, Novo Nordisk, Pfizer, Sanofi, Spark, and Takeda. Lizheng Shi is principal investigator for the project sponsored by BioMarin. Er Chen is an employee and stockholder of BioMarin. Louis P. Garrison did not receive funding for the research carried out in this work. However, he has received honoraria from BioMarin, serving on general advisory board on projects in development. He has also received funding from Pfizer and uniQure to speak on hemophilia, and he has been a consultant to numerous other life sciences companies, including Gilead, Roche GSK, BMS, Merck, and others. Milad Karimi is an employee and stockholder of BioMarin. Henry Mead was an employee and stockholder of BioMarin at the time of the study; he is now a consultant for BioBridges.

: Tulane University’s Human Research Protection office conducted an expedited review and approved the study protocol in accordance with appropriate research regulations. The study was conducted in accordance with the ethical standards of the 1964 Declaration of Helsinki and its later amendments.

: The authors would like to thank all patients who participated in this study. Their contributions were essential to the success of this research.