which is being evaluated in a phase 1/2 study for the treatment of patients with Alpha-1 Antitrypsin deficiency [AATD]. Initial results from this early-stage study are expected to be released in 2025.

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to hepatocellular carcinoma. The disease remains asymptomatic until its final ...

The recent conference, `Alpha 1-antitrypsin deficiency and other conformational diseases', held in Airlie, Virginia, USA (27–30 June, 2000) focused on some of the common pathways by which cells ...