METHODS OF DOSING ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENSE MUTATION SUPPRESSIVE THERAPY Russian patent published in 2012 - IPC A61K31/4245 A61P11/00 A61P21/00 A61P43/00 

Abstract RU 2462247 C2

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

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RU 2 462 247 C2

Authors

Khiravat Samit

Miller Langdon

Dates

2012-09-27Published

2007-10-11Filed