COMPOSITIONS AND METHODS FOR TREATING LYSOSOMAL DISORDERS

摘要

PROBLEM TO BE SOLVED: To provide compositions and methods for treating lysosomal disorders.SOLUTION: The invention provides a composition and a method for treating lysosomal disorders using a class of substituted imidazole derivatives or compounds. In some aspects of the invention, the chemical compound of Formula 1 or 1a (such as Formula 1b or 2) is administered to a subject suffering from lysosomal storage disease. The lysosomal storage disease is characterized by defect in glycosphingolipid synthesis. In some embodiments, the defect is present in synthesis of neutral glycosphingolipid. In other embodiments, the disease is characterized by a defect in ceramide synthesis. The disease may be Gaucher's disease or Fabry's disease.