Duchenne is a muscle wasting disease that occurs primarily in males and is caused by a deficiency of the dystrophin protein. There are many types of genetic mutations that can cause Duchenne, and NS-050/NCNP-03, an antisense oligonucleotide, is being developed to treat patients with confirmed gene mutations amenable to exon 50 skipping therapy. NS-050/NCNP-03 skips part of the genetic information of the dystrophin gene and produces a functional dystrophin protein with a slightly shorter chain length, which is expected to have the effect of suppressing muscle function deterioration.
It is not available yet, the company is preparing clinical trials of NS-050/NCNP-03 in patients with Duchenne in Japan and the United States.

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