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Acetyl-Leucine slows disease progression in lysosomal storage disorders

Abstract:

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 enantiomers acetyl-L-leucine and acetyl-D-leucine in symptomatic Npc1-/- mice and observed improvement in ataxia with both individual enantiomers and acetyl-DL-leucine. When acetyl-DL-leucine and ace...

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Publication status:
Published
Peer review status:
Peer reviewed

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Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
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Name:
Wellcome Trust
Grant:
202834/Z/16/Z
Publisher:
Oxford University Press
Journal:
Brain Communications More from this journal
Volume:
3
Issue:
1
Article number:
fcaa148
Publication date:
2020-09-15
Acceptance date:
2020-07-21
DOI:
ISSN:
2632-1297
Language:
English
Keywords:
Pubs id:
1120572
Local pid:
pubs:1120572
Deposit date:
2020-07-21

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