Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes

• Main Authors: Chotirmall, Sanjay Haresh, Al-Alawi, Mazen, McEnery, Thomas, McElvaney, Noel Gerard
• Other Authors: Lee Kong Chian School of Medicine (LKCMedicine)
• Format: Article
• Language: English
• Published: 2015
• Subjects: DRNTU::Science::Medicine::Pharmacy::Pharmaceutical technology
• Online Access: https://hdl.handle.net/10356/103341; http://hdl.handle.net/10220/25999
• Institution: Nanyang Technological University

Alpha-1 antitrypsin (AAT) deficiency remains an underrecognized genetic disease with predominantly pulmonary and hepatic manifestations. AAT is derived primarily from hepatocytes; however, macrophages and neutrophils are secondary sources. As the natural physiological inhibitor of several proteases,...