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    Glucosylsphingosine is a potential fluid-based biomarker of lysosomal dysfunction in Cln3Δex7/8 mice

    Glucosylsphingosine is a potential fluid-based biomarker of lysosomal dysfunction in Cln3Δex7/8 mice by Hallie Wald, Stephanie Cicalese, Lihang Yao, Nathan Hatcher, Weiwei Luo, Xiaolan Shen, Xiaoli Ping, Brie Culp, Dan Metzger, Mark Ault, Christian Nunes, Mali Cosden, Sarah Jinn, Jason Uslaner, Sean Smith, Jacob Marcus, Robert Drolet

    Published 2025-10-01

    CLN3 disease is a rare fatal juvenile neurodegenerative lysosomal storage disease. Challenges in tracking underlying disease biology have hindered the identification of effective therapeutic targets and the ability to execute clinical trials in this rare disease. While diagnostic biomarkers are read...

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