USE OF ARIMOCLOMOL IN ACTIVATING CLEAR GENE EXPRESSION AS TREATMENT FOR LYSOSOMAL STORAGE DISORDERS

原文（中国語）

The present disclosure provides methods of treating lysosomal storage disorders such as Niemann-Pick Disease, Type-C, by administering a therapeutically effective amount of arimoclomol in combination with miglustat. The present disclosure further provides methods of removing lysosomal cholesterol by administering the same.