Methods and compositions for increasing [alpha] -L-iduronidase activity in the central nervous system

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专利权人:

アーマジェン・インコーポレイテッド

发明人:

ウィリアム・エム・パードリッジ,ルーベン・ジェイ・ボアド

申请号：

JP2014156837

公开号：

JP6412731B2

申请日:

2014.07.31

申请国别(地区):

JP

年份:

2018

代理人:

摘要：

Provided herein are methods and compositions for treating a subject suffering from a deficiency in ±-L-Iduronidase in the CNS. The methods include systemic administration of a bifunctional fusion antibody comprising an antibody to a human insulin receptor and an ±-L-Iduronidase. A therapeutically effective systemic dose is based on the specific CNS uptake characteristics of human insulin receptor antibody-±-L-Iduronidase fusion antibodies as described herein.

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