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ANALOGUES OF BICALUTAMIDE OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS
ANALOGUES OF BICALUTAMIDE OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS
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机译:比卡洛胺或(S)-比卡洛胺的类似物作为促溶酶活性化合物用于溶酶体贮积病或糖原异生的治疗
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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.
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