Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F15%3A10312449" target="_blank" >RIV/00216208:11110/15:10312449 - isvavai.cz</a>
Alternative codes found
RIV/00064165:_____/15:10312449
Result on the web
—
DOI - Digital Object Identifier
—
Alternative languages
Result language
angličtina
Original language name
Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
Original language description
Fabry disease (FD) is a rare, X-linked glycosphingolipid storage disorders caused by mutations in the GLA gene (location chromosome Xq22.1). The resulting functional deficiency in the alpha-galactosidase A enzyme (EC 3.2.1.22) leads to accumulation of glycosphingolipids, especially globotriaosylceramide (Gb3), in lysosomes.
Czech name
—
Czech description
—
Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FA - Cardiovascular diseases including cardio-surgery
OECD FORD branch
—
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
Drug Design, Development and Therapy
ISSN
1177-8881
e-ISSN
—
Volume of the periodical
9
Issue of the periodical within the volume
neuveden
Country of publishing house
GB - UNITED KINGDOM
Number of pages
10
Pages from-to
3435-3444
UT code for WoS article
—
EID of the result in the Scopus database
—