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Open-label safety, tolerability, and pharmacokinetics study of eteplirsen in very young DMD patients amenable to exon 51 skipping
2016-000951-29Phase 2Unknown
- Sponsor
- Sarepta Therapeutics
- Start
- Apr 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 2, open-label, single-arm trial of eteplirsen in Duchenne muscular dystrophy (DMD), sponsored by Sarepta Therapeutics. Primary endpoint: Safety and tolerability of eteplirsen. Status: unknown.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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