New developments in biomarkers and clinical drug development in alpha‐1 antitrypsin deficiency‐related liver disease

R Loomba, G Clark, J Teckman… - Alimentary …, 2024 - Wiley Online Library
Background Alpha‐1 antitrypsin liver disease (AATLD) occurs in a subset of patients with
alpha‐1 antitrypsin deficiency. Risk factors for disease progression and specific …

Fazirsiran for adults with alpha-1 antitrypsin deficiency liver disease: a phase 2 placebo controlled trial (SEQUOIA)

VC Clark, C Strange, P Strnad, AJ Sanchez, P Kwo… - Gastroenterology, 2024 - Elsevier
Background & Aims Homozygous ZZ alpha-1 antitrypsin (AAT) deficiency produces mutant
AAT (Z-AAT) proteins in hepatocytes, leading to progressive liver fibrosis. We evaluated the …