Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial.

Michael, Mini; Groothoff, Jaap W; Shasha-Lavsky, Hadas; Lieske, John C; Frishberg, Yaacov; Simkova, Eva; Sellier-Leclerc, Anne-Laure; Devresse, Arnaud; Guebre-Egziabher, Fitsum; Bakkaloglu, Sevcan A; Mourani, Chebl; Saqan, Rola; Singer, Richard; Willey, Richard; Habtemariam, Bahru; Gansner, John M; Bhan, Ishir; McGregor, Tracy; Magen, Daniella

Michael, Mini; Groothoff, Jaap W; Shasha-Lavsky, Hadas; Lieske, John C; Frishberg, Yaacov; Simkova, Eva; Sellier-Leclerc, Anne-Laure; Devresse, Arnaud; Guebre-Egziabher, Fitsum; Bakkaloglu, Sevcan A; Mourani, Chebl; Saqan, Rola; Singer, Richard; Willey, Richard; Habtemariam, Bahru; Gansner, John M; Bhan, Ishir; McGregor, Tracy; Magen, Daniella.

Afiliación

Michael M; Division of Pediatric Nephrology, Department of Pediatrics, Texas Children's Hospital/Baylor College of Medicine, Houston, Texas. Electronic address: mmichael@bcm.edu.
Groothoff JW; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
Shasha-Lavsky H; Pediatric Nephrology Unit, Galilee Medical Center, Azrieli Faculty of Medicine, Bar Ilan University, Nahariya, Israel.
Lieske JC; Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota.
Frishberg Y; Division of Pediatric Nephrology, Shaare Zedek Medical Center, Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Simkova E; Nephrology - Medical Affairs, Al Jalila Children's Hospital, Dubai, United Arab Emirates.
Sellier-Leclerc AL; Hôpital Femme Mère Enfant en Centre d'Investigation Clinique, Institut National de la Santé et de la Recherche Médicale (INSERM), Hospices Civils de Lyon, ERKnet, Bron, France.
Devresse A; Division of Nephrology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
Guebre-Egziabher F; Nephrology and Renal Function Unit, Edouard Herriot Hospital, Hospices Civils de Lyon, INSERM 1060, Lyon, France.
Bakkaloglu SA; Department of Pediatric Nephrology, Faculty of Medicine, Gazi University, Ankara, Turkey.
Mourani C; Department of Pediatrics, Hôtel-Dieu de France Hospital, Beirut, Lebanon.
Saqan R; Pharmaceutical Research Center, Jordan University of Science and Technology, Irbid, Jordan.
Singer R; Renal Service, Canberra Health Services, Garran, ACT, Australia.
Willey R; Alnylam Pharmaceuticals, Cambridge, Massachusetts.
Habtemariam B; Alnylam Pharmaceuticals, Cambridge, Massachusetts.
Gansner JM; Alnylam Pharmaceuticals, Cambridge, Massachusetts.
Bhan I; Alnylam Pharmaceuticals, Cambridge, Massachusetts.
McGregor T; Alnylam Pharmaceuticals, Cambridge, Massachusetts.
Magen D; Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa, Israel.

Am J Kidney Dis ; 81(2): 145-155.e1, 2023 Feb.

Article en En | MEDLINE | ID: mdl-35843439

Asunto(s)

Hiperoxaluria Primaria; Hiperoxaluria; Enfermedades Renales; Adolescente; Adulto; Niño; Preescolar; Femenino; Humanos; Lactante; Recién Nacido; Masculino; Persona de Mediana Edad; Adulto Joven; Hiperoxaluria Primaria/complicaciones; Enfermedades Renales/complicaciones; Oxalatos

Palabras clave

Lumasiran; RNA interference (RNAi); adverse events; anti-drug antibodies; cardiac dysfunction; efficacy; glycolate; hemodialysis; kidney disease; nephrocalcinosis; pediatric; pharmacodynamics; pharmacokinetics; phase 3 clinical trial; plasma oxalate (POx); primary hyperoxaluria type 1 (PH1); safety; systemic oxalosis; urinary oxalate (UOx)

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Hiperoxaluria / Hiperoxaluria Primaria / Enfermedades Renales Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged / Newborn Idioma: En Revista: Am J Kidney Dis Año: 2023 Tipo del documento: Article

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