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Long-term open-label extension of BMN 351 in Duchenne muscular dystrophy amenable to exon 51 skipping
2024-511656-41-00Phase 2Authorised
- Sponsor
- BioMarin Pharmaceutical
- Start
- Apr 2026
- Modality
- Gene Therapies, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 5
01
Overview
Phase 2, open-label, single-arm trial of BMN-351 in Duchenne muscular dystrophy (DMD), sponsored by BioMarin Pharmaceutical. Primary endpoint: Safety and tolerability. Planned start Apr 2026; status: authorised.
02
Drugs
BMN-351
03
Conditions
Duchenne muscular dystrophy (DMD)
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