Acetyl-Leucine slows disease progression in lysosomal storage disorders

Acetyl-Leucine slows disease progression in lysosomal storage disorders

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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 en...

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Detalhes bibliográficos
Principais autores:	Kaya, E, Smith, DA, Smith, C, Morris, L, Fineran, P, Morten, KJ, Poulton, J, Frances Platt
Formato:	Journal article
Idioma:	English
Publicado em:	Oxford University Press 2020

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