BICALUTAMIDE ANALOGS OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN THE TREATMENT OF A LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS

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...

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Main Authors FARRERA-SINFREU, JOSEP, GORT MAS, LAURA, MATALONGA BORREL, LESLIE, FERRER MONTIEL, ANTONIO, PONSATI OBIOLS, BERTA, RIBES RUBIO, ANTONIA, PASCUAL MARTINEZ, ROBERTO
Format Patent
LanguageEnglish
Published 05.02.2021
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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.
Bibliography:Application Number: MY2016PI00977