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AVI-4658 dose-ranging study in exon 51-skippable Duchenne muscular dystrophy
ISRCTN28347032Phase 2Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Feb 2009
- 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 2, open-label, single-arm trial of eteplirsen in Duchenne muscular dystrophy (DMD), sponsored by Sarepta Therapeutics. Primary endpoint: Safety of escalating doses of AVI-4658. Started Feb 2009; status: completed.
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Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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