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SRP-4045 and SRP-4053 in Duchenne Muscular Dystrophy
2024-514698-23-00Phase 3Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Jul 2024
- 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
- 16
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Casimersen (SRP-4045), Golodirsen (SRP-4053) and Placebo (3 arms). Primary endpoint: Change from Baseline at Week 96 in 4-step ascend velocity. Started Jul 2024; status: completed.
02
Drugs
casimersengolodirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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