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Alpha1-antitrypsin Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Alpha1-antitrypsin Deficiency trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Alpha1-antitrypsin Deficiency trials you may qualify forThis is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) du…
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examinin…
This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and eff…
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(…
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal versio…
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the…
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outco…
This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standar…