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24-week randomized double-blind placebo-controlled trial of drisapersen in ambulant Duchenne muscular dystrophy with 72-week open-label extension
2014-005296-81Phase 3Completed
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Nov 2015
- 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 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Drisapersen with Placebo/Drisapersen (2 arms). Primary endpoint: Efficacy (primary). Started Nov 2015; status: completed.
02
Drugs
drisapersen
03
Conditions
Duchenne muscular dystrophy (DMD)
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