COMPOSITION CONTAINING ANTISENSE OLIGONUCLEOTIDE AND ITS USE FOR THE TREATMENT OF DUSCHENNE MUSCULAR DYSTROPHY Russian patent published in 2023 - IPC C12N15/113 A61K31/7088 A61P21/04 

FIELD: biotechnology.

SUBSTANCE: invention relates to a method of treatment of Duchenne muscular dystrophy, including intravenous administration of a pharmaceutical composition containing an antisense oligomer consisting of a nucleotide sequence complementary to a sequence consisting of nucleotides in positions 36 to 56 from the 5'-end of exon 53 of the gene human dystrophin, or a pharmaceutically acceptable salt or hydrate thereof. The antisense oligomer or a pharmaceutically acceptable salt or hydrate thereof is administered intravenously to a human patient once a week at a dose of 40 mg/kg/week inclusive to 80 mg/kg/week inclusive.

EFFECT: effective treatment of Duchenne muscular dystrophy.

25 cl, 16 dwg, 29 tbl, 10 ex