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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Main Authors FARRERA-SINFREU, JOSEP, GORT MAS, LAURA, MATALONGA BORREL, LESLIE, FERRER MONTIEL, ANTONIO, PASCUAL MARTÍNEZ, ROBERTO, PONSATI OBIOLS, BERTA, RIBES RUBIÓ, ANTONIA
Format Patent
LanguageEnglish
French
Published 02.07.2015
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary: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. La présente invention concerne une thérapie pour les maladies de surcharge lysosomale et la glycogenèse par traitement avec des composés qui promeuvent l'exocytose, de préférence l'exocytose lysosomale. Le traitement des cellules de patients affectés par différents troubles de surcharge lysosomale avec des composés activant l'exocytose conduit à une réduction de l'accumulation de substrat toxique dans les lysosomes, permettant ainsi le traitement, la prévention et le soulagement des symptômes d'un grand nombre de troubles de surcharge lysosomale.
Bibliography:Application Number: WO2014EP78745