compound, pharmaceutical composition, unit dosage form, and methods for modulating premature translational termination or nonsense-mediated mrna deterioration in a cell, for treating or preventing a genetic disorder or ameliorating one or more symptoms associated with the or manifestations of a genetic disease, to treat, prevent or ameliorate cancer or one or more symptoms associated with or manifestations of cancer, and to treat, prevent or ameliorate one or more symptoms associated with or manifestations of a disease

摘要

"COMPOUND, PHARMACEUTICAL COMPOSITION, UNIT DOSAGE FORM, AND METHODS FOR MODULATING PREMATURE TRANSLATION TERMINATION OR DETERIORATION OF MRNA MEASURED IN A CELL, TO TREAT OR PREVENT A GENETIC OR HONEY SUGAR OR HAZARDOUS DISEASE A GENETIC DISEASE, TO TREAT, AVOID OR IMPROVE CANCER OR ONE OR MORE SYMPTOMS ASSOCIATED WITH OR CANCER MANIFESTATIONS, AND TO TREAT, PREVENT OR IMPROVE SYMPTOMS ASSOCIATED WITH OR MANIFESTATIONS ". Novel 1,2,4-oxadiazole benzoic acid compounds, methods of use and pharmaceutical compositions comprising a 1,2,4-oxadiazole benzoic acid derivative are described. Methods include methods of treating or preventing an ameliorated disease by modulating premature translational termination or sensitized-mediated mRNA deterioration, or amelioration of one or more symptoms associated with it.