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Phase III open-label extension study of viltolarsen in ambulant boys with Duchenne muscular dystrophy amenable to exon 53 skipping
CTR20233077Phase 3Active
- Sponsor
- Nippon Shinyaku
- Start
- Oct 2023
- 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
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Overview
Phase 3, open-label, single-arm trial of viltolarsen in Duchenne muscular dystrophy (DMD), sponsored by Nippon Shinyaku. Primary endpoints: Vital signs, Physical examination and 5 more. Started Oct 2023; status: active.
02
Drugs
viltolarsen
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Conditions
Duchenne muscular dystrophy (DMD)
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