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Article

Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema.

Orphanet journal of rare diseases - 12 Apr 2011

Blanco Ignacio, Lara Beatriz, de Serres Frederick

Abstract excerpt

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 <11 μmol/L). However, the compassionate use of augmentation therapy in recent years has proven outstanding efficacy in small cohorts...

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Identifiers

PMID: 21486454
DOI: 10.1186/1750-1172-6-14
PMCID: PMC3094201

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