DMD CLINICAL THERAPIES II

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Duchenne muscular dystrophy (DMD) is an X-linked inherited progressive disease, caused by a mutation in the dystrophin gene. Exon skipping therapy uses a synthesized antisense oligonucleotide designed to skip the specific exon to change out-of-frame to in-frame in the dystrophin mRNA, thereby inducing the expression of functional dystrophin protein. NS-065/NCNP-01 is a novel morpholino oligomer discovered by NCNP and Nippon Shinyaku Co., Ltd. NS-065/NCNP-01 targets exon 53, to serve as effective treatment for DMD patients with deletion of exons 43-52, 45-52, 48-52, 49-52, 50-52 or 52 of dystrophin gene.

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