Plasma protein metabolism disease

This Phase 1 trial tested WVE-006, an RNA editing drug, in 47 healthy volunteers to see if it is safe and how the body processes it. The study is the first step toward a possible treatment for Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disea…

This study tested a new way to give Alpha-1 protein therapy by injecting it under the skin instead of into a vein. Seventeen adults with Alpha-1 Antitrypsin Deficiency received either a low or high dose weekly for eight weeks. The main goal was to check safety and how the drug mo…

This Phase 2 study tested a drug called fazirsiran in 40 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause liver disease. The drug is designed to lower levels of a harmful protein (Z-AAT) in the blood. Researchers measured safety and how much the prot…

This study tested a drug called fazirsiran in 16 people with alpha-1 antitrypsin deficiency, a genetic condition that can cause liver damage. The drug aims to reduce harmful protein buildup in the liver. Researchers measured changes in liver protein levels and safety over 24 to 4…

This study tested a simple, home-based breathing technique called the Active Cycle of Breathing Technique (ACBT) with augmented reality support in 50 adults with Alpha-1 antitrypsin deficiency. Participants did the breathing exercises twice daily and walked at least 5000 steps pe…

This early-stage study tested the safety and how the body processes a new drug called ARO-AAT in 45 healthy adults. The goal was to see if the drug is safe and to measure its levels in the blood. This research is a first step toward a possible treatment for Alpha-1 Antitrypsin De…

This study looked at how well the Pfizer COVID-19 vaccine triggers an immune response in 196 adults with weakened immune systems (due to conditions like kidney transplant, cancer, or HIV) compared to healthy adults. Researchers measured antibody levels and their ability to neutra…

This study looks at genetic differences in children with alpha-1 antitrypsin deficiency, a rare inherited condition that can cause liver disease. Researchers will compare gene patterns between children who develop liver problems and those who do not, using blood samples from abou…