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METHODS AND COMPOSITIONS FOR TREATMENT OF GLYCOGEN STORAGE DISEASES AND GLYCOGEN METABOLISM DISORDERS
专利权人:
Valerion Therapeutics; LLC
发明人:
ARMSTRONG, Dustin, D.
申请号:
EP15806109
公开号:
EP3154570A4
申请日:
2015.06.12
申请国别(地区):
EP
年份:
2017
代理人:
摘要:
The present disclosure provides for compositions comprising a chimeric polypeptide comprising a polypeptide effective for treating glycogen storage disease and an internalizing moiety that promotes delivery into cells. In certain embodiments, the polypeptide effective for treating glycogen storage disease is an acid alpha-glucosidase (GAA), a laforin, an amyloglucosidase (AGL), a malin, or an alpha amylase. The present disclosure also provides for methods for decreasing glycogen accumulation in cells or for treating glycogen storage diseases, including Forbes-Cori Disease, Andersen Disease, von Gierke Disease, Pompe Disease, and Lafora Disease, comprising administering the chimeric polypeptide disclosed herein.
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