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Phase 3 double-blind placebo-controlled study of viltolarsen in ambulant boys with Duchenne muscular dystrophy amenable to exon 53 skipping
jRCT2080224893Phase 3Completed
- Sponsor
- Nippon Shinyaku
- Start
- Sep 2019
- 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
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Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Viltolarsen with Placebo (2 arms). Primary endpoint: Change in TTSTAND velocity from baseline. Started Sep 2019; status: completed.
02
Drugs
viltolarsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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