BICALUTAMID ANALOGUE OR (S) -BICALUTAMID AS EXOCYTOSE ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISEASE OR GLYCOGENOSE

摘要

SUMMARY The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds leads to a decrease in the accumulation of toxic substrate in the lysosomes, thus allowing the treatment, prevention and relief of the symptoms of many lysosomal storage disorders.