发明名称 |
BICALUTAMIDE ANALOGS OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN THE TREATMENT OF A LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS |
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. |
申请公布号 |
PH12016501168(A1) |
申请公布日期 |
2016.07.18 |
申请号 |
PH12016501168 |
申请日期 |
2016.06.16 |
申请人 |
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 |
分类号 |
A61K31/00;A61K31/167 |
主分类号 |
A61K31/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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