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A METHOD FOR THE TREATMENT OF POMPE DISEASE USING 1- DEOXYNOJIRIMYCIN DERIVATIVES
A METHOD FOR THE TREATMENT OF POMPE DISEASE USING 1- DEOXYNOJIRIMYCIN DERIVATIVES
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机译:用1-去氧智联霉素衍生物治疗庞氏病的方法
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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 1-deoxynojirimycin derivative is substituted at the N or Cl position. Combination therapy with replacement α-glucosidase gene or enzyme is also provided.
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