Valerie ASKANAS,Anna NOGALSKA,Carla DAGOSTINO,William King ENGEL
申请号：
US14160081
公开号：
US20140206763A1
申请日:
2014.01.21
申请国别(地区):
US
年份:
2014
代理人:
摘要：
Disclosed is a method of improving lysosomal activity in a patient in need thereof including administering to the patient an effective amount of phenylbutyrate, preferably 4-phenylbutyrate in the form of at least one of sodium phenylbutyrate or glycerol phenylbutyrate. Also disclosed a method of improving lysosomal activity or ameliorating a pathological phenotype in a cell having impaired autophagy comprising treating the cell with phenylbutyrate, preferably 4-phenylbutyrate in the form of at least one of NaBP or GPB. The phenylbutyrate is generally administered in the form of a pharmaceutical composition including the phenylbutyrate and at least one carrier.
