Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels

Sinbaglustat is a brain-penetrating small molecule that inhibits the non-lysosomal glucocerebrosidase (GBA2) and, with lower potency, glucosylceramide synthase (GCS). Sinbaglustat has passed clinical phase I. Our preclinical study assessed its efficacy in a transgenic mouse model of GM1 gangliosidos...

ver descrição completa

Detalhes bibliográficos

Principais autores:	Rouven Wannemacher, Lorna Jubran-Rudolf, Isabel Zdora, Eva Leitzen, Karl Rohn, Virginie Sippel, Christoph Paschen, Peter Blattmann, Wolfgang Baumgärtner, Ingo Gerhauser, Michel Alexander Steiner
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2025-06-01
coleção:	Neurobiology of Disease
Assuntos:	β-galactosidase deficiency Brain GM1 gangliosidosis Substrate reduction therapy Sinbaglustat Axonal damage
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S0969996125001330
Tags:	Adicionar Tag Sem tags, seja o primeiro a adicionar uma tag!