Acetyl-leucine slows disease progression in lysosomal storage disorders

Acetyl-dl-leucine is a derivative of the branched chain amino acid leucine. In observational clinical studies, acetyl-dl-leucine improved symptoms of ataxia, in particular in patients with the lysosomal storage disorder, Niemann-Pick disease type C1. Here, we investigated acetyl-dl-leucine and its e...

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Detalles Bibliográficos
Autores principales: Kaya, Ecem, Smith, David A, Smith, Claire, Morris, Lauren, Bremova-Ertl, Tatiana, Cortina-Borja, Mario, Fineran, Paul, Morten, Karl J, Poulton, Joanna, Boland, Barry, Spencer, John, Strupp, Michael, Platt, Frances M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7954382/
https://www.ncbi.nlm.nih.gov/pubmed/33738443
http://dx.doi.org/10.1093/braincomms/fcaa148

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7954382/
https://www.ncbi.nlm.nih.gov/pubmed/33738443
http://dx.doi.org/10.1093/braincomms/fcaa148

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