ANALOGUES DE BICALUTAMIDE OU (S)-BICALUTAMIDE COMME COMPOSES ACTIVANT L'EXOCYTOSE DESTINES AU TRAITEMENT DE TROUBLE DE STOCKAGE LYSOSOMAL OU DE GLYCOGENESE

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专利权人:

BCN PEPTIDES; S.A.

发明人:

FARRERA-SINFREU, JOSEP,MATALONGA BORREL, LESLIE,GORT MAS, LAURA,PASCUAL MARTINEZ, ROBERTO,FERRER MONTIEL, ANTONIO,RIBES RUBIO, ANTONIA,PONSATI OBIOLS, BERTA

申请号：

CA2934772

公开号：

CA2934772A1

申请日:

2014.12.19

申请国别(地区):

CA

年份:

2015

代理人:

摘要：

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.

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