Goal 2: Reduce Human Disease

Clinical trial of AAT augmentation therapy in PI MZ subjects

Although severely alpha-1 antitrypsin deficiency (PI ZZ) individuals are rare, PI MZ subjects comprise approximately 3% of the US population. Mounting evidence suggests that PI MZ is a significant risk factor for COPD. A clinical trial of COPD exacerbation frequency using AAT augmentation therapy in PI MZ subjects with moderate-to-severe COPD could provide a rationale for AAT augmentation therapy (or small molecule neutrophil elastase inhibitors) in PI MZ subjects.

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Idea No. 1594