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 (S)-bicalutamide or its analogues, which promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis acti...

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Bibliographic Details
Main Authors Ponsati Obiols, Berta, Ribes Rubió, Antonia, Pascual Martínez, Roberto, Matalonga Borrel, Leslie, Farrera-Sinfreu, Josep, Gort Mas, Laura, Ferrer Montiel, Antonio
Format Patent
LanguageEnglish
Published 24.04.2020
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary:The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with (S)-bicalutamide or its analogues, which 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: NZ20140720499