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Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy

Trial Profile

Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 20 Aug 2020

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At a glance

  • Drugs Viltolarsen (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Registrational; Therapeutic Use
  • Sponsors Nippon Shinyaku
  • Most Recent Events

    • 13 Aug 2020 According to an Nippon Shinyaku media release, the U.S. Food & Drug Administration (FDA) has approved VILTEPSO (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. DMD is a progressive muscle disease that primarily occurs in boys due to a genetic mutation in the dystrophin gene.
    • 03 Oct 2018 Results published in the Nippon Shinyaku Media Release
    • 03 Oct 2018 According to a Nippon Shinyaku media release, the results were presented at The 23rd International Annual Congress of the World Muscle Society 2018.
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