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Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
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Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
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Journal Article
Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
2022
Overview
Severe AAT deficiency is caused by a mutation in
SERPINA1
, which encodes AAT. Mutant AAT protein accumulates in the liver and can lead to liver disease. This phase 2 trial of an agent targeting
SERPINA1
messenger RNA showed a reduction in levels of mutant AAT protein in affected persons.
