| Autor: |
Djalila Mekahli, Max C. Liebau, Melissa A. Cadnapaphornchai, Stuart L. Goldstein, Larry A. Greenbaum, Mieczyslaw Litwin, Tomas Seeman, Franz Schaefer, Lisa M. Guay-Woodford |
| Jazyk: |
angličtina |
| Rok vydání: |
2023 |
| Předmět: |
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| Zdroj: |
BMC Nephrology, Vol 24, Iss 1, Pp 1-11 (2023) |
| Druh dokumentu: |
article |
| ISSN: |
1471-2369 |
| DOI: |
10.1186/s12882-023-03072-x |
| Popis: |
Abstract Purpose 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. Methods Both trials are of multinational, multicenter, open-label design. Age range at enrollment is 28 days to |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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