ALPORT SYNDROME TREATMENT OPTIONS Russian patent published in 2023 - IPC A61K31/7125 A61P13/12 

FIELD: medicine, therapy, nephrology.

SUBSTANCE: group of inventions is intended for the treatment of Alport syndrome. A method for treating Alport’s syndrome includes administering to a patient with Alport’s syndrome two or more doses of the modified oligonucleotide. The modified oligonucleotide is composed of 19 linked nucleosides and has the structure of 5'-AECSATCSAGTCSTGAUSAAGCSTAE-3' (SEQ ID NO: 3) where the nucleosides without a subsequent subscript are β-D-deoxyribonucleosides, the nucleosides followed by the subscript E are 2-MOE-nucleosides, the nucleosides followed by the subscript S are S-CET-nucleosides and every inter-nucleoside bond is a phosphothioate inter-nucleoside bond. Wherein the dose of the said oligonucleotide is 1,5 mg/kg, which is administered at a frequency of two weeks between doses. In other embodiments, options are provided for treating Alport syndrome in a patient and a method for reducing the level of decline in kidney function over time in a patient with Alport syndrome.

EFFECT: improving kidney function, reducing or slowing further progression of renal fibrosis, delaying the onset of end stage renal disease, delaying time to dialysis, delaying time to kidney transplantation, prolonging the lifespan of the subject.

32 cl, 3 ex