Methods for treating autosomal dominant hypercholesterolemia associated with PCSK9 gain-of-function mutations

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专利权人:

Inc.;Regeneron Pharmaceuticals

发明人:

Swergold, Gary,Mellis, Scott,Sasiela, William J.

申请号：

AU2014274077

公开号：

AU2014274077B2

申请日:

2014.05.29

申请国别(地区):

AU

年份:

2019

代理人:

摘要：

The present invention provides methods for treating autosomal dominant hypercholesterolemia (ADH). According to certain embodiments, the ADH is caused by or associated with a gain-of- function mutation (GOFm) in a gene encoding PCSK9. The present invention therefore includes methods comprising selecting a patient who carries a GOFm in one or both alleles of the PCSK9 gene, and administering to the patient a pharmaceutical composition comprising a PCSK9 inhibitor. In certain embodiments, the PCSK9 inhibitor is an anti-PCSK9 antibody such as the exemplary antibody referred to herein as mAb316P.

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