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Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children.
Sas, David J; Magen, Daniella; Hayes, Wesley; Shasha-Lavsky, Hadas; Michael, Mini; Schulte, Indra; Sellier-Leclerc, Anne-Laure; Lu, Jiandong; Seddighzadeh, Ali; Habtemariam, Bahru; McGregor, Tracy L; Fujita, Kenji P; Frishberg, Yaacov.
Afiliação
  • Sas DJ; Division of Pediatric Nephrology and Hypertension, Mayo Clinic, Rochester, MN. Electronic address: sas.david@mayo.edu.
  • Magen D; Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa, Israel.
  • Hayes W; Department of Paediatric Nephrology, Great Ormond Street Hospital, London, United Kingdom.
  • Shasha-Lavsky H; Pediatric Nephrology Unit, Galilee Medical Center, Nahariya, Israel.
  • Michael M; Division of Pediatric Nephrology, Department of Pediatrics, Texas Children's Hospital/Baylor College of Medicine, Houston, TX.
  • Schulte I; Department of Pediatric Nephrology, University of Bonn, Bonn, Germany.
  • Sellier-Leclerc AL; Hôpital Femme Mère Enfant and Centre d'Investigation Clinique Inserm, Hospices Civils de Lyon, ERKnet, Bron, France.
  • Lu J; Alnylam Pharmaceuticals, Cambridge, MA.
  • Seddighzadeh A; Alnylam Pharmaceuticals, Cambridge, MA.
  • Habtemariam B; Alnylam Pharmaceuticals, Cambridge, MA.
  • McGregor TL; Alnylam Pharmaceuticals, Cambridge, MA.
  • Fujita KP; Alnylam Pharmaceuticals, Cambridge, MA.
  • Frishberg Y; Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem, Israel; Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel. Electronic address: Yaacovf@ekmd.huji.ac.il.
Genet Med ; 24(3): 654-662, 2022 03.
Article em En | MEDLINE | ID: mdl-34906487
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hiperoxalúria Primária / Terapêutica com RNAi Limite: Child, preschool / Humans / Infant Idioma: En Revista: Genet Med Assunto da revista: GENETICA MEDICA Ano de publicação: 2022 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hiperoxalúria Primária / Terapêutica com RNAi Limite: Child, preschool / Humans / Infant Idioma: En Revista: Genet Med Assunto da revista: GENETICA MEDICA Ano de publicação: 2022 Tipo de documento: Article