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Authors
Svoboda, Melissa D.
Kuntz, Nancy
Leon-Astudillo, Carmen
Byrne, Barry J.
Krueger, Jena
Kwon, Jennifer M.
Sieburg, Cory
Castro, Diana
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Funding
Funding for this research was provided by:
Genentech
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Article History
Received: 17 January 2025
Accepted: 5 June 2025
First Online: 7 July 2025
Declarations
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: Institutional review board approval was obtained or waived from the following institutions:1. Baylor College of Medicine/CHRISTUS Children’s Hospital (San Antonio, TX): the Baylor College of Medicine IRB approval was obtained and informed consent was provided by the parents or legal guardians.2. University of Florida College of Medicine (Gainesville, FL): University of Florida IRB waived consent. Consent for release of records were obtained from caregivers.3. Helen DeVos Children’s Hospital (Grand Rapids, MI): Corewell Health IRB approval (REF #2023–047) and ICF/HIPPA wavier was obtained.4. Ann & Robert H. Lurie Children’s Hospital of Chicago/Northwestern University Feinberg School of Medicine (Chicago, IL): Northwestern University IRB approval (REF #2022–5667) and informed consent was obtained from the parents or legal guardians.5. University of Wisconsin-Madison School of Medicine and Public Health (Madison, WI): the University of Wisconsin-Madison IRB waived consent.6. Neurology Rare Disease Center (Denton, TX): the institution granted the study exemption from IRB review. Informed consent was obtained from the parents or legal guardians.All methods and procedures were carried out in accordance with relevant guidelines and regulations. Furthermore, the study also adheres to the 2013 Helsinki Declarations.
: Not applicable.
: MDS participates in registry studies with Novartis and Genentech and a clinical study with Biohaven; NK serves on medical advisory boards for Argenx, Astellas, Biogen, BioMarin, FibroGen, Genentech, Novartis and Sarepta and has received compensation for educational talks on gene therapy from Sarepta; JK is a principal site investigator for clinical trials sponsored by Novartis, Biohaven, FibroGen, Genentech and Scholar Rock and serves on medical advisory boards for Astellas. JMK is a principal site investigator for sponsored clinical trials by Novartis and Scholar Rock in SMA; DC has received funding from Genentech, ReveraGen, Edgewise and Biohaven and consulting fees from Arugula Science; CL-A, BB and CS declare that they have no competing interests.
