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

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