Benjamin Mugrage,Gary Lee,Xiaoxiang Zhu,Robert Boyd,Kamlesh Sheth
申请号：
US14876018
公开号：
US20160051528A1
申请日:
2015.10.06
申请国别(地区):
US
年份:
2016
代理人:
摘要：
The present invention provides a method for increasing the activity of a mutant or wild-type α-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The I-deoxynojirimycin derivative is substituted at the N or C1 position. Combination therapy with replacement α-glucosidase gene or enzyme is also provided.
