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BMN 044 long-term extension in Duchenne muscular dystrophy
2015-003681-87Phase 2Completed
- 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 2, open-label, single-arm trial of PRO044 in Duchenne muscular dystrophy (DMD), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: Long-term safety of BMN 044 and Long-term efficacy of BMN 044. Started Nov 2015; status: completed.
02
Drugs
PRO044
03
Conditions
Duchenne muscular dystrophy (DMD)
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