| abstract |
The present invention relates to a lysosomal storage disease caused by an enzyme defect whose activity is not directly related to the presence of the lysosomal BMP cofactor, such as glycogen storage disease, gangliosidosis, neuronal ceroid lipofuscinosis, Cerebral tendon cholesterol, Wolman's disease, cholesteryl ester storage disorder, glycosaminoglycan metabolism disorder, mucopolysaccharidosis, glycoprotein metabolism disorder, mucolipidosis, aspartylglucosamineuria, fucoside storage disease, mannosidosis, and sialidosis type II The present invention relates to a bioactive agent that can increase the intracellular concentration and / or activity of Hsp70. [Selection] Figure 12 |