Trial Profile
A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 08 Dec 2021
Price :
$35
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At a glance
- Drugs Viltolarsen (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions; Pharmacokinetics; Registrational; Therapeutic Use
- Sponsors NS Pharma
- 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.
- 26 May 2020 Results evaluating the safety, tolerability, and efficacy of viltolarsen, a novel antisense oligonucleotide, in participants with DMD amenable to exon 53 skipping published in the JAMA Neurology
- 26 May 2020 According to a NS Pharma Media Release, data from this study was published in JAMA Neurology.