Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels. - Wannemacher R; Jubran-Rudolf L; Zdora I; Leitzen E; Rohn K; Sippel V; Paschen C; Blattmann P; Baumgärtner W; Gerhauser I; Steiner MA (Neurobiology of disease, 2025)