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High-dose eteplirsen dose-finding study in exon 51 skippable Duchenne muscular dystrophy
2024-511492-15-00Phase 3Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Dec 2021
- 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
- 24
01
Overview
Phase 3, randomised, double-blind trial of eteplirsen in Duchenne muscular dystrophy (DMD) (3 arms), sponsored by Sarepta Therapeutics. Primary endpoints: Open-label dose-escalation safety, NSAA interim efficacy and 1 more. Started Dec 2021; status: completed.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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