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COMPOSITIONS DE DNJ PARENTÉRALES STABLES
专利权人:
Amicus Therapeutics; Inc.
发明人:
申请号:
EP14738292.3
公开号:
EP2943200B1
申请日:
2014.01.09
申请国别(地区):
EP
年份:
2019
代理人:
摘要:
A stable pharmaceutical composition that includes an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, and a buffer, wherein the stable pharmaceutical composition is capable of being parenterally administered to a human without deleterious health effects. Pompe disease is an example of a lysosomal storage disorder. Pompe disease is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). GAA metabolizes glycogen, a storage form of sugar used for energy, into glucose.
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