Elevated lysosomal pH contributes to altered sphingolipid signaling and defective photoreceptor outer segment (POS) phagocytosis in a stem cell-derived model of CLN3 Batten disease.

AT-GTX-502 - Amicus

Ankur Rakesh Dubey1; Sonal Dalvi1; Jimin Han1; Nathaniel Foley1; Fesahat Emira Dogrul1; Leah Grego1; Ruchira Singh1,2

Mar 26, 2025