| abstract |
The present invention provides a method for increasing the activity of an α-glucosidase enzyme and a method for treating Pompe disease. A method for increasing the activity of an α-glucosidase enzyme in a cell comprising contacting an effective amount of a formula 1-deoxynojirimycin derivative with the enzyme. [Wherein, R 1 and R 2 each represent H, or optionally substituted alkyl, etc., and at least one of R 1 and R 2 is not H] and is also the 1-deoxynojirimycin derivative A method of treating Pompe disease by administration of a chaperone small molecule compound. Combination therapy with a substituted α-glucosidase gene or enzyme is also provided. [Selection] Figure 1 |