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Trial intelligence / Profile preview
Long-term open-label extension study of BMN 044 in Duchenne muscular dystrophy amenable to exon 44 skipping
NL-OMON43638Phase 2Withdrawn
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Dec 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
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Overview
Phase 2, open-label trial of PRO044 in Duchenne muscular dystrophy (DMD) (2 arms), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: Long-term safety of BMN 044, 6-minute walk distance and 14 more. Planned start Dec 2015; status: withdrawn.
02
Drugs
PRO044
03
Conditions
Duchenne muscular dystrophy (DMD)
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