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BMN 053 exon 53 skipping study in ambulant boys with Duchenne muscular dystrophy
NL-OMON43770Phase 2Active, not recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Aug 2013
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 2, open-label trial of BMN-053 in Duchenne muscular dystrophy (DMD) (5 arms), sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Primary efficacy endpoint. Started Aug 2013; status: active, not recruiting.
02
Drugs
BMN-053
03
Conditions
Duchenne muscular dystrophy (DMD)
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