Inventors:
Wei Zheng - Potomac MD, US
Juan Jose Marugan - Gaithersburg MD, US
Ke Liu - Cambridge MD, US
Noel Terrence Southall - Chevy Chase MD, US
Christopher P. Austin - Potomac MD, US
Assignee:
The United States of America as Represented by the Secretary Department of HR - Rockville MD
International Classification:
A61K 31/355, A61K 31/724, A61K 45/06
Abstract:
This disclosure relates generally to the treatment of lysosomal storage disorders. Specifically, the disclosure relates to a novel use of delta tocopherol in the treatment of diseases and conditions related to lysosomal storage disorders. Included in the present disclosure is a method for the modulation of cholesterol recycling. Further, the disclosure relates to conditions such as Niemann-Pick type C disease, Farber disease, Niemann-Pick type A disease, Wolman disease and Tay Sachs disease. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol in combination with cyclodextrin to a patient in need thereof.