Plasma protein metabolism disease

This study tests whether inhaling alpha-1 antitrypsin (AAT) daily can slow lung function loss in people with Alpha-1 deficiency and moderate-to-severe lung disease. 220 adults will receive either the drug or a placebo for two years, then all will receive the drug for two more yea…

This study tested an experimental drug called KRRO-110 in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause lung and liver damage. The goal was to check safety and how the drug moves through the body. However, the trial w…

This study tested a new drug called belcesiran in people with a genetic condition called alpha-1 antitrypsin deficiency that causes liver damage. The goal was to see if the drug is safe and can lower harmful protein levels in the liver. The trial was stopped early, so results are…

This early-phase study tested a drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can lead to lung and liver damage. The main goal was to check safety and how the drug affects levels of a key protein in the b…

This study tested an experimental drug called VX-864 in 14 people with a severe form of alpha-1 antitrypsin deficiency (PiZZ genotype). The goal was to see if the pill could raise protective protein levels in the blood and reduce harmful buildup in the liver. The trial was termin…