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
Title | Year | Author | Number |
---|---|---|---|
METHOD FOR PRODUCING FUNCTIONAL PROTEIN FROM DNA HAVING NONSENSE MUTATIONS, AND TREATING ASSOCIATED DISORDERS | 2007 |
|
RU2462246C2 |
METHODS OF PRODUCING 1,2,4-OXADIAZOLEBENZOIC ACIDS | 2007 |
|
RU2495030C2 |
COMPOSITIONS AND METHODS OF TREATING ANAEMIA | 2014 |
|
RU2705206C2 |
CRYSTALLINE FORMS OF 3-[5-(2-FLUOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]BENZOIC ACID, AND USE THEREOF AND METHODS FOR PREPARING | 2007 |
|
RU2474577C2 |
ORAL COMPOSITIONS OF CARDIAC SARCOMERE INHIBITORS | 2020 |
|
RU2823999C2 |
COMPOSITIONS AND METHODS OF TREATING DISEASES AND CONDITIONS | 2015 |
|
RU2713886C2 |
COMBINATIONS | 2015 |
|
RU2715236C2 |
PHARMACEUTICAL COMPOSITION FOR PREVENTION OR TREATMENT OF DIABETES AND RELATED METABOLIC DISEASES | 2021 |
|
RU2809286C1 |
PHARMACEUTICALLY ACTIVE COMPOUNDS | 2013 |
|
RU2654942C2 |
PHARMACEUTICAL COMPOSITION FOR PREVENTION OR TREATMENT OF NON-ALCOHOLIC STEATHOEPATITIS | 2021 |
|
RU2803733C1 |
Authors
Dates
2012-09-27—Published
2007-10-11—Filed