COMPOUNDS AND METHODS FOR TREATING OR PREVENTING DISEASE CONDITIONS ASSOCIATED WITH ALPHA-1-ANTITRYPSIN
摘要
Pharmacological chaperone compounds and methods for the treatment of an individual having, or at risk of having, a disease condition associated with alpha-1-antitrypsin by using said compounds are disclosed. In particular, such methods are useful for the treatment 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. Also provided is an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.
申请公布号
WO2012051518(A3)
申请公布日期
2012.06.21
申请号
WO2011US56335
申请日期
2011.10.14
申请人
AMICUS THERAPEUTICS, INC.;SJOBERG, ERIC, RICHARD;LEE, GARY