Bicalutamide Analogs Or (S)-Bicalutamide As Exocytosis Activating Compounds For Use In The Treatment Of A Lysosomal Storage Disorder Or Glycogenosis

• 收藏
• 分享

专利权人:

发明人:

Josep FARRERA-SINFREU,Leslie MATALONGA BORREL,Laura GORT MAS,Roberto PASCUAL MARTÍNEZ,Antonio FERRER MONTIEL,Antonia RIBES RUBIÓ,Berta PONSATI OBIOLS

申请号：

US15102358

公开号：

US20160317489A1

申请日:

2014.12.19

申请国别(地区):

US

年份:

2016

代理人:

摘要：

SUMMARY 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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/