Clinical Effect and Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients With Cholesteryl Ester Storage Disease

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F13%3A10189900" target="_blank" >RIV/00216208:11110/13:10189900 - isvavai.cz</a>

Alternative codes found

RIV/00064165:_____/13:10189900

Result on the web

<a href="http://dx.doi.org/10.1002/hep.26289" target="_blank" >http://dx.doi.org/10.1002/hep.26289</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1002/hep.26289" target="_blank" >10.1002/hep.26289</a>

Result language

angličtina

Original language name

Clinical Effect and Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients With Cholesteryl Ester Storage Disease

Original language description

Cholesteryl ester storage disease (CESD), an inherited deficiency of lysosomal acid lipase (LAL), is an underappreciated cause of progressive liver disease with no approved therapy. Presenting features include dyslipidemia, elevated transaminases, and hepatomegaly. To assess the clinical effects and safety of the recombinant human LAL, sebelipase alfa, nine patients received four once-weekly infusions (0.35, 1, or 3 mgkg(-1)) in LAL-CL01, which is the first human study of this investigational agent. Patients completing LAL-CL01 were eligible to enroll in the extension study (LAL-CL04) in which they again received four once-weekly infusions of sebelipase alfa (0.35, 1, or 3 mgkg(-1)) before transitioning to long-term every-other-week infusions (1 or 3 mgkg(-1)). Sebelipase alfa was well tolerated, with mostly mild adverse events unrelated to sebelipase alfa. No antidrug antibodies were detected. Transaminases decreased in patients in LAL-CL01 and increased between studies. In seven pati

Czech name

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Czech description

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Type

J_x - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)

CEP classification

FG - Paediatrics

OECD FORD branch

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Project

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Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2013

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Hepatology

ISSN

0270-9139

e-ISSN

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Volume of the periodical

58

Issue of the periodical within the volume

3

Country of publishing house

US - UNITED STATES

Number of pages

8

Pages from-to

950-957

UT code for WoS article

000329284000016

EID of the result in the Scopus database

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