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A new brain‐penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease

Gaucher disease (GD), the most common lysosomal storage disorders, is caused by GBA gene mutations resulting in glycosphingolipids accumulations in various tissues, such as the brain. While suppressing glycosphingolipid accumulation is the central strategy for treating peripheral symptoms of GD, the...

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Detalles Bibliográficos
Autores principales:	Fujii, Takahiro, Tanaka, Yuta, Oki, Hideyuki, Sato, Sho, Shibata, Sachio, Maru, Takamitsu, Tanaka, Maiko, Onishi, Tomohiro
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	ORIGINAL ARTICLES
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293090/ https://www.ncbi.nlm.nih.gov/pubmed/34398463 http://dx.doi.org/10.1111/jnc.15492

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