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Open-label extension study of drisapersen in Duchenne muscular dystrophy
2015-001955-54Phase 3Terminated
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Jul 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, open-label, single-arm trial of drisapersen in Duchenne muscular dystrophy (DMD), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: Long-term safety and tolerability and Long-term efficacy. Started Jul 2015; status: terminated.
02
Drugs
drisapersen
03
Conditions
Duchenne muscular dystrophy (DMD)
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