Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases

A Wanner, SC Groft, JR Teagarden, J Krischer, BR Davis, CS Coffey, DH Hickam, J Teckman, DR Nelson, ML McCaleb, R Loomba, C Strange, RA Sandhaus, M Brantly, JM Edelman, Albert Farrugia

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