| 摘要 |
FIELD: medicine, pharmaceutics. ^ SUBSTANCE: invention refers to medicine and concerns a method of treating or regulating nonsense mutation(s) associated diseases: cystic fibrosis caused by a premature stop codon in mRNA coded by the CFTR gene, and Duchene muscular dystrophy caused by a premature stop codon in mRNA coded by the dystrophin gene. The method involves the introduction of the effective amount of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazole-3-yl]benzoic acid or its pharmaceutically acceptable salts, solvate or hydrate in the patient in need thereof within continued treatment wherein a first dose, a second dose and a third dose are introduced for 24 hours by formula 1X, 1X and 2X wherein X represents a dose of 18-22 mg/kg. ^ EFFECT: invention provides safe and effective dosing schemes of the drug preparations which reduce side effects and enables an optimal therapeutic effect. ^ 17 cl, 2 dwg, 8 tbl, 17 ex |
