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Publication | Open Access

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

DOIFull Paper Access

72

Citations

23

References

2021

Year

David J. Sas, Daniella Magen, Wesley Hayes, Hadas Shasha‐Lavsky, Mini Michael, Indra Schulte, Anne‐Laure Sellier‐Leclerc, Jiandong Lu, Ali Seddighzadeh, Bahru Habtemariam,
Genetics in Medicine

Abstract

Lumasiran showed rapid, sustained reduction in spot UOx:Cr and plasma oxalate and acceptable safety in patients aged <6 years with PH1, establishing RNA interference therapies as safe, effective treatment options for infants and young children.

References

YearCitations

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