Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
About this item
Full title
Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
Author / Creator
Publisher
Boston: Massachusetts Medical Society
Journal title
The New England journal of medicine, 2022-08, Vol.387 (6), p.514-524
Language
English
Formats
Publication information
Publisher
Boston: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Severe AAT deficiency is caused by a mutation in
SERPINA1
, which encodes AAT. Mutant AAT protein accumulates in the liver and can lead to liver disease. This phase 2 trial of an agent targeting
SERPINA1
messenger RNA showed a reduction in levels of mutant AAT protein in affected persons.
Alternative Titles
Full title
Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency
Authors, Artists and Contributors
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2681032670
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2681032670
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2205416
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