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 PASCUAL MARTÍNEZ, Roberto, PONSATI OBIOLS, Berta, RIBES RUBIÓ, Antonia, MATALONGA BORREL, Leslie, FERRER MONTIEL, Antonio, FARRERA-SINFREU, Josep, GORT MAS, Laura
Format Patent
LanguageEnglish
French
German
Published 16.10.2019
Subjects
HUMAN NECESSITIES
HYGIENE
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES
MEASURING
MEDICAL OR VETERINARY SCIENCE
PHYSICS
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
TESTING
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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: EP20140815747