Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial
| Autor: | Yaacov Frishberg, Wesley Hayes, Hadas Shasha-Lavsky, David J. Sas, Mini Michael, Anne-Laure Sellier-Leclerc, Julien Hogan, Richard Willey, John M. Gansner, Daniella Magen |
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| Jazyk: | angličtina |
| Rok vydání: | 2024 |
| Předmět: | |
| Zdroj: | Frontiers in Pediatrics, Vol 12 (2024) |
| Druh dokumentu: | article |
| ISSN: | 2296-2360 62092111 |
| DOI: | 10.3389/fped.2024.1392644 |
| Popis: | BackgroundPrimary hyperoxaluria type 1 (PH1) is a genetic disorder resulting in overproduction of hepatic oxalate, potentially leading to recurrent kidney stones, nephrocalcinosis, chronic kidney disease, and kidney failure. Lumasiran, the first RNA interference therapeutic approved for infants and young children, is a liver-directed treatment that reduces hepatic oxalate production. Lumasiran demonstrated sustained efficacy with an acceptable safety profile over 12 months in infants and young children (age 45 ml/min/1.73 m2 if ≥12 months old or normal serum creatinine if |
| Databáze: | Directory of Open Access Journals |
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