Acetyl-DL-leucine in Niemann-Pick type C A case series
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F15%3A10312692" target="_blank" >RIV/00216208:11110/15:10312692 - isvavai.cz</a>
Alternative codes found
RIV/00064165:_____/15:10312692
Result on the web
<a href="http://dx.doi.org/10.1212/WNL.0000000000002041" target="_blank" >http://dx.doi.org/10.1212/WNL.0000000000002041</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1212/WNL.0000000000002041" target="_blank" >10.1212/WNL.0000000000002041</a>
Alternative languages
Result language
angličtina
Original language name
Acetyl-DL-leucine in Niemann-Pick type C A case series
Original language description
Objective:To assess the effects of the modified amino acid acetyl-dl-leucine (AL) on cerebellar ataxia, eye movements, and quality of life of patients with Niemann-Pick type C (NP-C) disease.Methods:Twelve patients with NP-C disease were treated with AL3 g/d for 1 week and then with 5 g/d for 3 weeks with a subsequent washout period of 1 month. The Scale for the Assessment and Rating of Ataxia (SARA), the Spinocerebellar Ataxia Functional Index (SCAFI), the modified Disability Rating Scale (mDRS), EuroQol 5Q-5D-5L, and the visual analog scale (VAS) were administered. Measurements took place at baseline, after 1 month of therapy, and after 1 month of washout.Results:The SARA score changed from the baseline (median [SD, interquartile range]) of 10.8 (11.2, 8-24.6) to 7.0 (10.7, 5.6-19.6) on medication (difference: 3.8 points) and 10.5 (11.5, 7.1-23.9) after washout (difference: 3.5 points) (p = 0.000412; post hoc p = 0.003 between baseline and on medication, and on medication and washou
Czech name
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Czech description
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Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FH - Neurology, neuro-surgery, nuero-sciences
OECD FORD branch
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Result continuities
Project
—
Continuities
V - Vyzkumna aktivita podporovana z jinych verejnych zdroju
Others
Publication year
2015
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Neurology
ISSN
0028-3878
e-ISSN
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Volume of the periodical
85
Issue of the periodical within the volume
16
Country of publishing house
US - UNITED STATES
Number of pages
8
Pages from-to
1368-1375
UT code for WoS article
000363288600004
EID of the result in the Scopus database
2-s2.0-84944717615