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Eteplirsen multiple-dose placebo-controlled phase 2 study in ambulant Duchenne muscular dystrophy
2016-005000-26Phase 2
- 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 2, randomised, double-blind trial of eteplirsen in Duchenne muscular dystrophy (DMD) (4 arms), sponsored by Sarepta Therapeutics. Primary endpoint: Change from baseline in percentage of dystrophin-positive fibers.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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