FIELD: medicine.
SUBSTANCE: group of inventions refers to medicine and concerns an antisense compound for reducing expression of superoxide dismutase 1 (SOD-1), where antisense compound has the following formula: mCes Aeo Ges Geo Aes Tds Ads mCds Ads Tds Tds Tds mCds Tds Ads mCeo Aes Geo mCes Te (sequence of nitrogen bases SEQ ID NO: 725) or a pharmaceutically acceptable salt thereof. Group of inventions also relates to a pharmaceutical composition for reducing the expression of superoxide dismutase 1 (SOD-1) containing said antisense compound or a pharmaceutically acceptable salt thereof; method of treating or preventing SOD-1 related neurodegenerative disorder or SOD-1 related ALS, involving administering said antisense compound or its pharmaceutically acceptable salt.
EFFECT: group of inventions provides treating or preventing the SOD-1 related neurodegenerative disorder or SOD-1 related ALS.
17 cl, 19 ex, 71 tbl
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Authors
Dates
2019-10-30—Published
2015-04-01—Filed