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Phase 3 randomized double-blind placebo-controlled study of viltolarsen in ambulatory boys with Duchenne muscular dystrophy amenable to exon 53 skipping
CTR20213303Phase 3Active
- Sponsor
- Nippon Shinyaku
- Start
- Jan 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
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Viltolarsen with Placebo (2 arms). Primary endpoint: Time to stand from supine (TTSTAND) at Week 48. Started Jan 2022; status: active.
02
Drugs
viltolarsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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