Orphanet Journal of Rare Diseases (Apr 2011)
Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema
Abstract
Abstract Up to now alpha 1-antitrypsin (AAT) augmentation therapy has been approved only for commercial use in selected adults with severe AAT deficiency-related pulmonary emphysema (i.e. PI*ZZ genotypes as well as combinations of Z, rare and null alleles expressing AAT serum concentrations