GLYCOLIPIDS AS TREATMENT FOR DISEASE

摘要

This invention provides compounds, compositions, and methods for treating a disorder selected from cancer, hyperinsulinemia, hypoglycemia, hyperinsulinemia with hypoglycemia, atypical Parkinson's disease, Huntington's disease, multiple systems atrophy, GM3 synthase deficiency, GM2 synthase deficiency or tauopathy.

主权项

1. A method of treating a subject having a disorder associated with a ganglioside deficiency, said method comprising administering to the subject a therapeutic amount of a compound of the formula:wherein Z is O, S, C(R10)2 and NR2, X is H, D, —OR11, —NR11R12, —SR11 and —CHR11R12, and R10, R11 and R12 independently selected from H, D, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted heterocycloalkyl, —C(═B)R13, —C(═B)—Z—R13, —SO2R13, and —SO3 functional moieties; B, B′, and Z independently selected from O, NR14 or S; Y is selected from H, D, —OR15, —SR15, —NR15R16, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, and substituted or unsubstituted heterocycloalkyl moieties; R1, R2, R3, R4, R5, R6, R7, R8, R9, R13, R14 independently selected from H, D, —OR17, —NR17R18, —SR19, and —CHR19R20, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted heterocycloalkyl moieties; R15, R16, R17, R18, and R19 independently selected from substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted heterocycloalkyl moieties, —C(═B′)R20, —C(═B′)—Z—R20, —SO2R20, and —SO3 functional moieties; R20 is selected from H, D, —OR21, —SR21, —NR21R22, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted heterocycloalkyl moieties; R21 and R22 are independently selected from H, D, substituted or unsubstituted alkyl, substituted or unsubstituted heteroalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted heterocycloalkyl moieties; n is an integer from 1 to 30.