Fazirsiran ‘quite amazing’ in treatment of alpha-1 antitrypsin deficiency liver disease

Fazirsiran effectively reduced the production and accumulation of a protein that causes alpha-1 antitrypsin deficiency and subsequent liver fibrosis in a small subset of patients, according to data in The New England Journal of Medicine.“We had strong evidence from preclinical studies and the phase I that fazirsiran would knock down [alpha-1 antitrypsin] mRNA in the liver,” senior author Jeffrey Teckman, MD, director of pediatric gastroenterology and hepatology and professor of pediatrics and biochemistry at Saint Louis University School of Medicine, told Healio. “TheRead More