BMN-351
- Generic Name
- BMN-351
- Drug Type
- Biotech
- Background
BMN-351 is a fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNA. It is under investigation for the treatment of Duchenne Muscular Dystrophy (DMD).
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants with Duchenne Muscular Dystrophy
- First Posted Date
- 2024-02-28
- Last Posted Date
- 2024-12-05
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 18
- Registration Number
- NCT06280209
- Locations
- 🇳🇱
Leids Universitair Medisch Centrum, Leiden, Netherlands
🇹🇷Yeditepe University Kosuyolu Hospital, Istanbul, Turkey
🇬🇧Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
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