Alpha-1 antitrypsin deficiency (AATD ... This results in progressive liver disease secondary to AAT polymerization and accumulation, and chronic obstructive pulmonary disease (COPD) due to ...
The recent conference, `Alpha 1-antitrypsin deficiency and other conformational ... protein structures associated with conformational disease was a major focus of the meeting.
However, no approved therapies exist for AATD-related liver disease, often leading to ... Pi*ZZ AATD do not naturally produce wild-type alpha-1 antitrypsin (M-AAT) protein. Thus, detecting the ...
Liver diseases can be inherited (such as Wilson disease and alpha 1-antitrypsin deficiency) or acquired (for example hepatitis and liver cancer). In 2024, important studies highlighted the ongoing ...
Terry – who is 59 – was diagnosed with COPD – or, Chronic Obstructive Pulmonary Disease – four years ... predisposition to COPD due to alpha 1 antitrypsin deficiency.
In our in vivo portfolio, the BEAM-302 clinical program in alpha-1 antitrypsin deficiency is ... cell therapy for the treatment of sickle cell disease (SCD). Of these patients, eight have been ...
Achieved first-ever RNA editing in humans in RestorAATion-2 trial of WVE-006 (GalNAc-AIMer) in alpha-1 antitrypsin deficiency and announced three wholly owned ... that preserves healthy wild-type ...