Document is current

Article History

Received: 22 August 2025

Revised: 25 February 2026

Accepted: 12 March 2026

First Online: 30 March 2026

Competing interests

: JKB has nothing to disclose. TM became an employee of BioMarin Pharmaceutical, Inc., after the conduct of the trial. AS has nothing to disclose. HL has nothing to disclose. DG was Chief Medical Officer of LogicBio Therapeutics. MP was an employee of Alexion, AstraZeneca Rare Disease, and holds stock or stock options in AstraZeneca. MAK was a co-founder of LogicBio Therapeutics and served on the Board of Directors and Scientific Advisory Board, and as a consultant for LogicBio Therapeutics. FC is an employee of Alexion, AstraZeneca Rare Disease, and holds stock or stock options in AstraZeneca. ESS is an employee of Alexion, AstraZeneca Rare Disease, and holds stock or stock options in AstraZeneca. QL was an employee of Alexion, AstraZeneca Rare Disease, and holds stock or stock options in AstraZeneca. JV received research funding from LogicBio to conduct this study.

: The study was approved by the IRB at each participating study site (University of Pittsburgh [MOD20010208-002]; Vanderbilt University [201305]; Seattle Children’s Hospital [20203011]; Emory University [STUDY00002158]). All participants or their parents, guardians, or legal representatives provided written informed consent and assent. The study was performed in accordance with the current Good Clinical Practice Guideline of the International Council for Harmonisation and appropriate regulatory requirements. An independent data and safety monitoring board (DSMB) and sponsor medical monitor provided study oversight.