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Phase 3 placebo-controlled trial of SRP-4045 or SRP-4053 in Duchenne muscular dystrophy amenable to exon 45 or 53 skipping
MOH_2017-06-18_000531Phase 3Not yet recruiting
- Sponsor
- Sarepta Therapeutics
- Start
- Mar 2017
- 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 Standard treatment + SRP-4045, Standard treatment + SRP-4053 and Standard treatment + placebo (3 arms). Primary endpoint: Primary endpoint not reported. Planned start Mar 2017; status: not yet recruiting.
02
Drugs
casimersengolodirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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