The invention relates to 20-hydroxyecdysone and the derivatives thereof, for use in the treatment of genetic myopathies.

The invention relates to 20-hydroxyecdysone and the derivatives thereof, for use in the treatment of genetic myopathies.

Provided herein are spirolactone compounds of Formula I that are useful as inhibitors of ACC1 and/or ACC2. The spirolactone compounds described herein can be used for treating a disease or disorder associated with aberrant ACC1 and/or ACC2 activities, for example, non-alcoholic steatohepatitis (NASH), acne, obesity, diabetes, and cancer. Also provided herein are pharmaceutical compositions comprising the spirolactone compound of Formula I, or pharmaceutically acceptable salt thereof. ##STR00001##

Provided herein are spirolactone compounds of Formula I that are useful as inhibitors of ACC1 and/or ACC2. The spirolactone compounds described herein can be used for treating a disease or disorder associated with aberrant ACC1 and/or ACC2 activities, for example, non-alcoholic steatohepatitis (NASH), acne, obesity, diabetes, and cancer. Also provided herein are pharmaceutical compositions comprising the spirolactone compound of Formula I, or pharmaceutically acceptable salt thereof. ##STR00001##

A composition containing SUMO inhibitor, and belongs to the technical field of medicine includes FXR agonist and SUMO inhibitor. In activated hepatic stellate cells, the FXR agonist does not have an effect in inhibiting the activation of hepatic stellate cells. After the FXR agonist and the SUMO inhibitor are compounded according to the present invention, the activation of hepatic stellate cells can also be inhibited for those cells under activated state. Of note, the hepatic stellate cells of an individual with hepatic fibrosis symptoms have been in an activated state, therefore a good anti-fibrotic effect cannot be achieved by using the FXR agonist alone.

A composition containing SUMO inhibitor, and belongs to the technical field of medicine includes FXR agonist and SUMO inhibitor. In activated hepatic stellate cells, the FXR agonist does not have an effect in inhibiting the activation of hepatic stellate cells. After the FXR agonist and the SUMO inhibitor are compounded according to the present invention, the activation of hepatic stellate cells can also be inhibited for those cells under activated state. Of note, the hepatic stellate cells of an individual with hepatic fibrosis symptoms have been in an activated state, therefore a good anti-fibrotic effect cannot be achieved by using the FXR agonist alone.

A composition containing SUMO inhibitor, and belongs to the technical field of medicine includes FXR agonist and SUMO inhibitor. In activated hepatic stellate cells, the FXR agonist does not have an effect in inhibiting the activation of hepatic stellate cells. After the FXR agonist and the SUMO inhibitor are compounded according to the present invention, the activation of hepatic stellate cells can also be inhibited for those cells under activated state. Of note, the hepatic stellate cells of an individual with hepatic fibrosis symptoms have been in an activated state, therefore a good anti-fibrotic effect cannot be achieved by using the FXR agonist alone.

Phosphonium-based ionic conjugates (PBICs) are described. The PBICs each include a cationic binding partner comprising a phosphonium ion and an anionic binding partner comprising a pharmaceutically active compound, or prodrug, or derivative thereof. The conjugate can have at least one enhanced physiochemical, pharmacokinetic and/or therapeutic quality as compared to the pharmaceutically active compound when not provided in a PBIC. The phosphonium-containing cationic binding partner can also serve to enhance delivery of the anionic binding partner to the cytosol and/or the inner mitochondrial space. Methods of preparing the PBICs and using the PBICs to treat disease are also described.

Phosphonium-based ionic conjugates (PBICs) are described. The PBICs each include a cationic binding partner comprising a phosphonium ion and an anionic binding partner comprising a pharmaceutically active compound, or prodrug, or derivative thereof. The conjugate can have at least one enhanced physiochemical, pharmacokinetic and/or therapeutic quality as compared to the pharmaceutically active compound when not provided in a PBIC. The phosphonium-containing cationic binding partner can also serve to enhance delivery of the anionic binding partner to the cytosol and/or the inner mitochondrial space. Methods of preparing the PBICs and using the PBICs to treat disease are also described.

This invention relates to clear one-phase liquid formulation vehicles comprising lecithin, MCT, bile salt and water.