Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children

Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children

Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants and young children with PH1. This single-arm, open-label, phase 3 study evaluated lumasiran in patien...

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Veröffentlicht in:Genetics in medicine 2022-03, Vol.24 (3), p.654-662
Hauptverfasser: 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, Bacchetta, Justine, Baudouin, Véronique, Becker-Cohen, Rachel, Tzvi Behr, Shimrit, Ben-Shalom, Efrat, Berdaguer, Maria, Bockenhauer, Detlef, Cochat, Pierre, Coenen, Martin, Cramer, Carl H., Deschênes, Georges, Dossier, Claire, Doye, Emilie, Feldman, Liat Feraru, Hohenadel, Maximilian, Kaguelidou, Florentia, Zebegret, Irina Libinson, Lieske, John C., Maisin, Anne, Milliner, Dawn S., Plonsky Toder, Moran, Pollack, Shirley, Portefaix, Aurélie, Ranchin, Bruno, Rinat, Choni, Safdar, Adnan, Schalk, Gesa, Srivaths, Poyyapakkam R., Tran, Cheryl L., Van't Hoff, William, Weinbrand-Goichberg, Jenny, Weissman, Irith
Format: Artikel
Sprache:eng
Schlagworte:
Child, Preschool
Humans
Hyperoxaluria, Primary - complications
Hyperoxaluria, Primary - genetics
Hyperoxaluria, Primary - therapy
Infant
Infants
Lumasiran
PH1
RNA Interference
RNA, Small Interfering
RNAi
RNAi Therapeutics
Young children
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Beschreibung
Zusammenfassung:Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants and young children with PH1. This single-arm, open-label, phase 3 study evaluated lumasiran in patients aged 45 mL/min/1.73 m2, if aged ≥12 months, or normal serum creatinine, if aged
ISSN:1098-3600
1530-0366
DOI:10.1016/j.gim.2021.10.024