Compounds and method for treating or preventing disease conditions associated with alpha-1-antitrypsin
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United States of America Patent
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Jan 16, 2018
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Feb 23, 2015
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Oct 14, 2010
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Abstract
The present invention provides compounds and methods for the treatment of an individual having or at risk of having a condition associated with alpha-1-antitrypsin by using a pharmacological chaperone. In particular, such methods are useful for the treatment and/or prevention of lung disorders associated with alpha-1-antitrypsin deficiency as well as liver disorders associated with an excess of alpha-1-antitrypsin. Suitable pharmacological chaperones include peptides and low-molecular weight compounds. The present invention also provides an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.
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