π₯π₯ Fazirsiran in Alpha-1-antitrypsin deficiency #ILC2022 @NEJM by Dr Pavel Strnad
Phase II RCT, n=16
Sc Fazirsiran on w1, w4 & Q12wly:
β¬ Z-AAT in liver (all)
β¬ liver globule burden (all)
β LFT improvement (all)
β Fibrosis regression
#LiverTwitter @DrLoomba @MattiasMandorf1
Phase II RCT, n=16
Sc Fazirsiran on w1, w4 & Q12wly:
β¬ Z-AAT in liver (all)
β¬ liver globule burden (all)
β LFT improvement (all)
β Fibrosis regression
#LiverTwitter @DrLoomba @MattiasMandorf1
What is Fazirsiran?
β RNA interference (RNAi)
- synthetic RNA duplex bind to asialoglycoprotein
> degradation of AAT and Z-AAT mRNA
> β¬AAT and Z-AAT in liver
#LiverTwitter @EASLedu @EASLedu @aelsharkawy75 @jturnesv #ILC2022
β RNA interference (RNAi)
- synthetic RNA duplex bind to asialoglycoprotein
> degradation of AAT and Z-AAT mRNA
> β¬AAT and Z-AAT in liver
#LiverTwitter @EASLedu @EASLedu @aelsharkawy75 @jturnesv #ILC2022
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