Phase 2 single-arm study: Fazirsiran for liver disease associated with Alpha1-Antitrypsin deficiency.

Fazirsiran for Liver Disease Associated with Alpha₁-Antitrypsin Deficiency – New England Journal of Medicine (link to abstract – $ for full-text)

Commentary on Twitter

In this small trial in adults with ATT deficiency, fazirsiran was associated with a strong reduction of mutant AAT levels in the serum and liver and concurrent improvements in liver enzyme levels. #ILC2022 https://t.co/RibsKzOQfO pic.twitter.com/BnMcSyfVcq

— NEJM (@NEJM) June 25, 2022