Trial Profile
Exploratory study of NS 065/NCNP 01 in Duchenne muscular dystrophy
Status:
Completed
Phase of Trial:
Phase I
Latest Information Update: 07 Nov 2021
Price :
$35
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At a glance
- Drugs Viltolarsen (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions; First in man; Registrational
- Sponsors Nippon Shinyaku
- 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.
- 19 Apr 2018 According to a Nippon Shinyaku media release, registry of Muscular Dystrophy (Remudy) was utilized for patient recruitment in The Study.
- 19 Apr 2018 Results published in the Nippon Shinyaku Media Release