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 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 07.02.2023
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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Abstract 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.
AbstractList 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.
Author Ferrer Montiel, Antonio
Ponsati Obiols, Berta
Pascual Martínez, Roberto
Farrera-Sinfreu, Josep
Matalonga Borrel, Leslie
Gort Mas, Laura
Ribes Rubió, Antonia
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– fullname: Farrera-Sinfreu, Josep
– fullname: Gort Mas, Laura
– fullname: Ferrer Montiel, Antonio
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Snippet The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal...
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SubjectTerms 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
Title Bicalutamide analogs or (S)-bicalutamide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis
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