Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial

Autor: William van’t Hoff, David Erbe, Pierre Cochat, Daniella Magen, Patrick Haslett, Jaap W. Groothoff, Jiandong Lu, Yaacov Frishberg, Pushkal Garg, Georges Deschênes, Akshay Vaishnaw, Sandeep Talamudupula, Ulrike Lorch, Sally-Anne Hulton, Jérôme Harambat, Tracy L. McGregor, John C. Lieske, Dawn S. Milliner, Bahru A. Habtemariam

Publikováno v: Clinical Journal of the American Society of Nephrology
Clinical Journal of the American Society of Nephrology, American Society of Nephrology, 2021, pp.CJN.14730920. ⟨10.2215/cjn.14730920⟩
Clinical journal of the American Society of Nephrology, 16(7), 1025-1036. American Society of Nephrology
Clin J Am Soc Nephrol

International audience; BACKGROUND AND OBJECTIVES: In the rare disease primary hyperoxaluria type 1, overproduction of oxalate by the liver causes kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. Lumasiran, an RNA interference

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1739805a88aa63ffcc6bf79348807932
https://hal.archives-ouvertes.fr/hal-03279682