Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease

Sandhoff disease is a rare neurodegenerative lysosomal storage disease associated with the storage of GM2 ganglioside in late endosomes/lysosomes. Here, we explored the efficacy of acetyl-DL-leucine (ADLL), which has been shown to improve ataxia in observational studies in patients with Niemann–Pick...

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Detalles Bibliográficos
Autores principales: Kaya, Ecem, Smith, David A., Smith, Claire, Boland, Barry, Strupp, Michael, Platt, Frances M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7230825/
https://www.ncbi.nlm.nih.gov/pubmed/32276303
http://dx.doi.org/10.3390/jcm9041050

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7230825/
https://www.ncbi.nlm.nih.gov/pubmed/32276303
http://dx.doi.org/10.3390/jcm9041050

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