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Authors
Ziółkowska, Ewa A.
Pagán Torres, Nicole A.
Chen, Hsintsung
Williams, Letitia L.
Eultgen, Elizabeth M.
Nowacka, Agnieszka
Dearborn, Joshua T.
Nelvagal, Hemanth R.
Whiteman, Ineka T.
Platt, Frances M.
Cooper, Jonathan D.
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Funding
Funding for this research was provided by:
The Beyond Batten Disease Foundation (N/A)
Institutional support from the Department of Pediatrics, Washington University in St. Louis (N/A)
National Institutes of Health (NINDS R01 NS124655)
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Article History
Received: 23 September 2025
Accepted: 15 December 2025
First Online: 29 December 2025
Declarations
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: JDC has received research support from BioMarin Pharmaceutical Inc., Abeona Therapeutics Inc., REGENXBIO Inc. and Neurogene, and is a consultant for JCR Pharmaceuticals. FMP is an academic cofounder of IntraBio.
: All animal procedures were performed in accordance with National Institutes of Health (NIH) guidelines under protocols 2018 − 0215, 21–0292, and 24–0232 approved by the Institutional Animal Care and Use Committee (IACUC) at Washington University School of Medicine in St. Louis, MO.
