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

AT-GTX-502 - Amicus

http://www.ncbi.nlm.nih.gov/pubmed/40639691

Brie Culp; Christian Nunes; Dan Metzger; Hallie Wald; Jacob Marcus; Jason Uslaner; Lihang Yao; Mali Cosden; Mark Ault; Nathan Hatcher; Robert Drolet; Sarah Jinn; Sean Smith; Stephanie Cicalese; Weiwei Luo; Xiaolan Shen; Xiaoli Ping

Jul 11, 2025