Triheptanoin diet for adult polyglucosan body disease (APBD) treatment

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专利权人:

Baylor Research Institute

发明人:

Schiffmann, Raphael,Mochel, Fanny

申请号：

AU2011267894

公开号：

AU2011267894A1

申请日:

2011.06.13

申请国别(地区):

AU

年份:

2012

代理人:

摘要：

Compositions and methods for the treatment and management of adult polyglucosan body disease (APBD) are disclosed herein. The APBD patients studied in the present invention experienced stabilization of disease progression and limited functional improvement with dietary triheptanoin (C7TG). The amount of C7TG administered to the patient daily for 6-8 months was 1-2 g/kg/24 hrs. The present invention demonstrates, for the first time, the arrest of clinical deterioration with limited functional recovery in APBD with triheptanoin diet therapy.

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