Bicalutamide analogs or (S)-bi calutam ide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis

SUMMARY The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds thatpromote exocytosis, preferably lysosomal exocytosis. The treatment of cells frompatients affected by different lysosomal storage disorders with exocytosis activating compounds lea...

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Hauptverfasser: FARRERA-SINFREU, JOSEP, GORT MAS, LAURA, MATALONGA BORREL, LESLIE, FERRER MONTIEL, ANTONIO, PONSATI OBIOLS, BERTA, RIBES RUBIO, ANTONIA, PASCUAL MARTINEZ, ROBERTO
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Sprache:eng
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Zusammenfassung:SUMMARY The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds thatpromote exocytosis, preferably lysosomal exocytosis. The treatment of cells frompatients affected by different lysosomal storage disorders with exocytosis activating compounds leads to a decreaseinthe accumulation of toxic substrate in the lysosomes, thus allowingthe treatment,prevention and relief of the symptoms of many lysosomal storage disorders.