abstract |
A compound for use in a method of treating, preventing or controlling a disease associated with a lack of gene expression caused by the stop codon of the prematurus, wherein the compound is 3- [5- (2-fluoro-phenyl) - [1,2,4] oxadiazol-3-yl 1-benzoic acid or a pharmaceutically acceptable salt, solvate or hydrate thereof, wherein the disease is cystic fibrosis or Duchenne muscular dystrophy, the method comprising administering the compound in three daily doses, the first dose, the second dose and the third dose being administered in 24 doses. time period according to formula 1X, 1X, 2X, where X is 18-22 mg / kg and where each dose maintains a plasma level of 3- [5- (2-fluoro-phenyl) - [1,2,4] oxadiazole-3- yl] -benzoic acid greater than 2 μg / ml for at least 2 hours. The application contains 9 more claims. |