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 Antonio FERRER MONTIEL, Antonia RIBES RUBIÓ, Laura GORT MAS, Berta PONSATI OBIOLS, Roberto PASCUAL MARTÍNEZ, Josep FARRERA-SINFREU, Leslie MATALONGA BORREL
Format Patent
LanguageEnglish
Spanish
Published 08.09.2016
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 invención proporciona un tratamiento para las enfermedades de acúmulo lisosomal y las glucogenosis mediante compuestos que promueven la exocitosis, preferentemente la exocitosis lisosomal. El tratamiento de células de pacientes afectados por distintos tipos de enfermedades de acúmulo lisosomal con compuestos que promueven la exocitosis da lugar a una disminución del acúmulo tóxico de sustratos en los lisosomas y permite el tratamiento, prevención y alivio de los síntomas de muchos desórdenes de acúmulo lisosomal.
Bibliography:Application Number: MX20160008283