Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy
AssigneeNippon Shinyaku Co., Ltd.
InventorsTomonori UNO, Takashi NATSUKAWA, Youichi EGAWA, Youhei SATOU
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Abstract
The present invention relates to a composition containing an antisense oligonucleotide, and the use thereof to treat Duchenne muscular dystrophy. The present invention particularly relates to the above-described composition that is effective for the treatment of Duchenne muscular dystrophy when it is administered at a dose for the treatment, and the use thereof.
Claims18
1-22. (canceled)
A pharmaceutical composition for treating a human patient with Duchenne muscular dystrophy (DMD) comprising an antisense oligomer, a pharmaceutically acceptable salt thereof, or a hydrate thereof in a concentration of between 2.5 mg/ml inclusive and 500 mg/ml inclusive,
- wherein the antisense oligomer consists of a base sequence complementary to a sequence consisting of nucleotides at positions 36 to 56 from the 5′-terminus of exon 53 of a human dystrophin gene.
Dependencies are inferred from the claim text.