摘要 |
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. |
申请人 |
THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES;ZHENG, WEI;MARUGAN, JUAN JOSE;LIU, KE;SOUTHALL, NOEL TERRENCE;AUSTIN, CHRISTOPHER P. |
发明人 |
ZHENG, WEI;MARUGAN, JUAN JOSE;LIU, KE;SOUTHALL, NOEL TERRENCE;AUSTIN, CHRISTOPHER P. |