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The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease

The result's identifiers

  • Result code in IS VaVaI

    <a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11120%2F07%3A00000484" target="_blank" >RIV/00216208:11120/07:00000484 - isvavai.cz</a>

  • Result on the web

  • DOI - Digital Object Identifier

Alternative languages

  • Result language

    angličtina

  • Original language name

    The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease

  • Original language description

    Purpose: This 10-week study was conducted to determine the pharmacokinetics of varying doses of agalsidase alfa and evaluate the effect of dose and dosing frequency on plasma Gb(3) levels. Methods: Eighteen adult male Fabry patients, naive to enzyme replacement therapy, were randomized to one of five regimens: 0.1, 0.2, or 0.4 mg/kg weekly; 0.2 mg/kg every other week (the approved dose); or 0.4 mg/kg every other week. Intravenous infusion rate was 0.1 mg/kg per 20 minutes. Plasma Gb(3) levels were assessed at baseline and periodically during the study. Results: The mean half-life was 56-76 minutes, and the mean volume of distribution at steady state was 17%-18% of body weight, with no significant association between dose and half-life, clearance, or volume of distribution at steady state. The area under the curve was linearly proportional to the dose from 0.1 to 0.4 mg/kg. Baseline average plasma Gb(3) was 9.12 +/- 2.61 nmol/mL and after 10 weeks of treatment was significantly reduced

  • Czech name

    Farmakologie podávání alfa-galaktosidázy alfa v různých léčebných režimech v rámci enzym-substituční léčby Fabryho chorob

  • Czech description

    Autoři popisují farmakologii podávání alfa-galaktosidázy alfa v různých léčebných režimech v rámci enzym-substituční léčby Fabryho chorob

Classification

  • Type

    J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)

  • CEP classification

    FR - Pharmacology and apothecary chemistry

  • OECD FORD branch

Result continuities

  • Project

  • Continuities

    V - Vyzkumna aktivita podporovana z jinych verejnych zdroju

Others

  • Publication year

    2007

  • Confidentiality

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

Data specific for result type

  • Name of the periodical

    Genetetics in Medicine

  • ISSN

    1098-3600

  • e-ISSN

  • Volume of the periodical

    9

  • Issue of the periodical within the volume

    8

  • Country of publishing house

    US - UNITED STATES

  • Number of pages

    6

  • Pages from-to

    504-509

  • UT code for WoS article

  • EID of the result in the Scopus database