Alpha-1 antitrypsin (AAT) deficiency is a rare, inherited disorder that can lead to liver disease in children and liver and lung disease in adults. Despite progress in the development of treatments for lung complications, there are no available therapies specifically for AAT deficiency-associated liver disease (AATLD).
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References
National Institutes of Health, U.S. National Library of Medicine, Lister Hill National Center for Biomedical Communications. Genetics Home Reference: Alpha-1 Antitrypsin Deficiency. https://ghr.nlm.nih.gov/condition/alpha-1-antitrypsin-deficiency. Published Sept. 2019. Accessed Jan. 2020.
De Serres F, Blanco I. Role of Alpha-1 antitrypsin in human health and disease. Journal of Internal Medicine. 2014; 276: 311-335.