Fazirsiran for Liver Disease Associated with Alpha 1 -Antitrypsin Deficiency.
Pavel StrnadMattias MandorferGourab ChoudhuryWilliam GriffithsChristian TrautweinRohit LoombaThomas SchluepTing ChangMin YiBruce D GivenJames C HamiltonJavier San MartinJeffery H TeckmanPublished in: The New England journal of medicine (2022)
In this small trial, fazirsiran was associated with a strong reduction of Z-AAT concentrations in the serum and liver and concurrent improvements in liver enzyme concentrations. (Funded by Arrowhead Pharmaceuticals; AROAAT-2002 ClinicalTrials.gov number, NCT03946449.).