Alpha-1 is a microcosm of the rare disease world. The challenges we face, late diagnoses, inconsistent care, and unaffordable ...

Alpha-1-antitrypsin is a so-called protease inhibitor, a type of enzyme inhibitor. It is produced in the liver but exerts its effects in the lungs, where it regulates immune cell activity. This ...

Korro Bio, Inc. announced that its investigational drug KRRO-110 has received orphan drug designation from the FDA for treating Alpha-1 Antitrypsin Deficiency (AATD). This designation highlights ...

In a nutshell: • Hereditary disease alpha-1-antitrypsin deficiency: researchers have discovered why some patients remain healthy despite a genetic defect, while others develop severe liver ...