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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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Bibliographische Detailangaben
Veröffentlicht in:J Clin Med
Hauptverfasser: Kaya, Ecem, Smith, David A., Smith, Claire, Boland, Barry, Strupp, Michael, Platt, Frances M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI 2020
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7230825/
https://ncbi.nlm.nih.gov/pubmed/32276303
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm9041050
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