FIELD: medicine, pharmaceutics.
SUBSTANCE: claimed group of inventions relates to field of medicine. Claimed is method of relieving at least one symptom of spinal muscular atrophy in subject, which includes introduction to subject of antisense compound, containing antisense oligonucleotide, complementary to intron 7 of pre-mRNA, coding human SMN2. Claimed is application of such antisense compound in production of medication for relieving or treatment of at least one symptom of spinal muscular atrophy in subject.
EFFECT: claimed group of inventions provides effective means and methods for relieving spinal muscular atrophy in subject.
13 cl, 13 dwg, 14 tbl, 14 ex
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