Use of Lysosomal Acid Lipase for treating Lysosomal Acid Lipase Deficiency in patients

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专利权人:

Synageva Biopharma Corp.

发明人:

Quinn, Anthony

申请号：

AU2011314293

公开号：

AU2011314293A1

申请日:

2011.09.09

申请国别(地区):

AU

年份:

2013

代理人:

摘要：

The present invention provides treating LAL deficiency (e.g. Wolman's disease, CESD) comprising administering to a mammal a therapeutically effective amount o lysosomal acid lipase with an effective dosage frequency. Methods of improving growth and liver function, increasing LAL tissue concentration, and increasing LAL activity in a human patient suffering from LAL deficiency are also provided

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