Bicalutamide Analogs Or (S)-Bicalutamide 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 that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds l...

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Main Authors GORT MAS Laura, FARRERA-SINFREU Josep, PONSATI OBIOLS Berta, RIBES RUBIÓ Antonia, MATALONGA BORREL Leslie, FERRER MONTIEL Antonio, PASCUAL MARTÍNEZ Roberto
Format Patent
LanguageEnglish
Published 03.11.2016
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
TESTING
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Summary: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: US201415102358