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

• 主要な著者: Yaacov Frishberg (著者), Wesley Hayes (著者), Hadas Shasha-Lavsky (著者), David J. Sas (著者), Mini Michael (著者), Anne-Laure S (著者), Julien Hogan (著者), Richard Willey (著者), John M. Gansner (著者), Daniella Magen (著者)
• フォーマット: 図書
• 出版事項: Frontiers Media S.A., 2024-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

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...