FIELD: medicine.
SUBSTANCE: invention relates to the field of medicine and is intended for the treatment of spinal muscular atrophy (SMA). Method includes the introduction, through a bolus injection into the intrathecal space of a human subject, of an antisense compound containing an antisense oligonucleotide consisting of 18 linked nucleosides, where each internucleoside oligonucleotide bond is a phosphorothioate bond, where each oligonucleotide nucleoside is 2'-MOE nucleoside and where the administration of an antisense compound enhances the incorporation of exon 7 into SMN2 mRNA transcripts in a human subject.
EFFECT: invention provides an effective treatment for SMA types I, II and III.
12 cl, 13 dwg, 14 tbl, 14 ex
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