PHENYLBUTYRATE FOR TREATMENT OF SPORADIC INCLUSION-BODY MYOSITIS AND DISORDERS RELATING TO AUTOPHAGY IMPAIRMENT OR AMYLOID BETA 42 ACCUMULATION

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.

主权项

1. A method for treating s-inclusion body myositis, s-IBM, or ameliorating one or more pathological phenotypes of s-IBM comprising administering to a patient in need thereof an effective amount of phenylbutyrate.