Journal article
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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Funding
Bibliographic Details
- Publisher:
- Oxford University Press Publisher's website
- Journal:
- Brain Communications Journal website
- Volume:
- 3
- Issue:
- 1
- Article number:
- fcaa148
- Publication date:
- 2020-09-15
- Acceptance date:
- 2020-07-21
- DOI:
- ISSN:
-
2632-1297
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
1120572
- Local pid:
- pubs:1120572
- Deposit date:
- 2020-07-21
Terms of use
- Copyright holder:
- Kaya et al.
- Copyright date:
- 2020
- Rights statement:
- © The Author(s) (2020). Published by Oxford University Press on behalf of the Guarantors of Brain. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.
- Licence:
- CC Attribution (CC BY)
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