Abstract: Disclosed herein are novel uses of ADMDP stereoisomers or their derivatives for the manufacture of a medicament for treating Pompe disease. Accordingly, the present disclosure provides a method of treating Pompe disease in a subject. The method includes the step of, administering to the subject a therapeutically effective amount of a compound of formula (I), or a salt, an ester or a solvate thereof, wherein R1 and R2 are independently H or alkyl optionally substituted by —NH2 or —OH, so as to ameliorate, alleviate mitigate and/or prevent symptoms associated with the Pompe disease. According to certain embodiments of the present disclosure, the compound of formula (I) may serve a stabilizer of ?-glucosidase via preventing its denaturalization of deactivation.

Abstract: An adeno-associated virus (AAV) vector is used for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The AAV vector is directly injected into human brain through a stereotactic technique. Activity of AADV is thus recovered for solving the problem of movement disorder.