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Phase II open-label study of viltolarsen versus natural history control in ambulatory and non-ambulatory boys with Duchenne muscular dystrophy amenable to exon 53 skipping
CTR20213302Phase 2Active
- Sponsor
- Nippon Shinyaku
- Start
- Jul 2022
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
A Phase 2 trial of viltolarsen in Duchenne muscular dystrophy (DMD), sponsored by Nippon Shinyaku. Started Jul 2022; status: active.
02
Drugs
viltolarsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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Competitive context
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