A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F61989592%3A15110%2F15%3A33162165" target="_blank" >RIV/61989592:15110/15:33162165 - isvavai.cz</a>
Result on the web
<a href="http://www.nejm.org/doi/full/10.1056/NEJMoa1501365#t=article" target="_blank" >http://www.nejm.org/doi/full/10.1056/NEJMoa1501365#t=article</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1056/NEJMoa1501365" target="_blank" >10.1056/NEJMoa1501365</a>
Alternative languages
Result language
angličtina
Original language name
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
Original language description
Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.
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
FG - Paediatrics
OECD FORD branch
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Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
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
New England Journal of Medicine
ISSN
0028-4793
e-ISSN
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Volume of the periodical
11
Issue of the periodical within the volume
3737
Country of publishing house
US - UNITED STATES
Number of pages
21
Pages from-to
1010-1020
UT code for WoS article
000360959000006
EID of the result in the Scopus database
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