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Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease

Autosomal recessive polycystic kidney disease (ARPKD) is a hereditary condition characterized by massive kidney enlargement and developmental liver defects. Potential consequences during childhood include the need for kidney replacement therapy (KRT). We report the design of 2 ongoing clinical trial...

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Hauptverfasser: Mekahli, Djalila (VerfasserIn) , Liebau, Max C. (VerfasserIn) , Cadnapaphornchai, Melissa A. (VerfasserIn) , Goldstein, Stuart L. (VerfasserIn) , Greenbaum, Larry (VerfasserIn) , Litwin, Mieczysław (VerfasserIn) , Seeman, Tomas (VerfasserIn) , Schaefer, Franz (VerfasserIn) , Guay-Woodford, Lisa M. (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 13 February 2023
In: BMC nephrology
Year: 2023, Jahrgang: 24, Pages: 1-11
ISSN:1471-2369
DOI:10.1186/s12882-023-03072-x
Online-Zugang:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s12882-023-03072-x
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Verfasserangaben:Djalila Mekahli, Max C. Liebau, Melissa A. Cadnapaphornchai, Stuart L. Goldstein, Larry A. Greenbaum, Mieczyslaw Litwin, Tomas Seeman, Franz Schaefer and Lisa M. Guay-Woodford
Beschreibung
Zusammenfassung:Autosomal recessive polycystic kidney disease (ARPKD) is a hereditary condition characterized by massive kidney enlargement and developmental liver defects. Potential consequences during childhood include the need for kidney replacement therapy (KRT). We report the design of 2 ongoing clinical trials (Study 204, Study 307) to evaluate safety, tolerability, and efficacy of tolvaptan in children with ARPKD.
Beschreibung:Gesehen am 19.04.2023
Beschreibung:Online Resource
ISSN:1471-2369
DOI:10.1186/s12882-023-03072-x

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