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：

AU2015218427

公开号：

AU2015218427A1

申请日:

2015.08.25

申请国别(地区):

AU

年份:

2015

代理人:

摘要：

#$%^&*AU2015218427A120150910.pdf#####H:\imt\Intenvovcn\NRPortbl\DCC\AMT\8305733_l.docx-24/08/2015 ABSTRACT The present invention provides treating LAL deficiency (e.g. Wolmans disease, CESD) comprising administering to a mammal a therapeutically effective amount of 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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