Medicament for treatment of duchenne muscular dystrophy

Details Medicament for treatment of duchenne muscular dystrophy Medicament for treatment of duchenne muscular dystrophy

C - Chemistry, Metallurgy

07

H

C07H 21/00 (2006.01) A61K 9/19 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) A61P 21/00 (2006.01) C12N 15/11 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2319149

Antisense oligonucleotides comprising a nucleotide sequence complementary to the nucleotide sequence of SEQ ID NO:1 or NO:2 are disclosed. The antisense oligonucleotides are used for treatment of specific types of Duchenne muscular dystrophy which is attributed to a change in number of the nucleotides composing one or more exons adjacent to exon 43 or 53, respectively, in human dystrophin mRNA, wherein said change is due to deletion of one or more nucleotides from the normal nucleotide sequence for said exons, wherein the net of said change in number of the nucleotides is expressed as a reduction of (3 X N+ 1) nucleotides, wherein N is zero or a natural number.

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