Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of ...

Alpha-1 antitrypsin deficiency, AATD, is an inherited disease affecting the lung, liver, and, rarely, skin. Alpha-1 antitrypsin, AAT, is a protease inhibitor of the proteolytic enzyme elastase.

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Least-squares mean percent differences in serum Z-AAT ...

As part of the multicenter National Heart, Lung, and Blood Institute registry of patients with severe deficiency of alpha 1-antitrypsin with 1,129 enrollees, this report describes measures undertaken ...

Please provide your email address to receive an email when new articles are posted on . The FDA granted orphan drug designation for KB408 for the treatment of alpha-1 antitrypsin deficiency, according ...

BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results from ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by the misfolding and accumulation of the mutant variant of alpha-1 antitrypsin (AAT) within hepatocytes, which limits its ...

NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD) NTLA-3001 ...