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BMN 351 phase 1/2 dose-escalation study in Duchenne muscular dystrophy
2023-506737-30-00Phase 2Completed
- Sponsor
- BioMarin Pharmaceutical
- Start
- Mar 2024
- 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 endpoints: Incidence of adverse events, serious adverse events, adverse events of special interest, physical examination findings, safety laboratory parameters, ECG parameters, and echocardiography, Physical examination and 2 more. Started Mar 2024; status: completed.
02
Drugs
BMN-351
03
Conditions
Duchenne muscular dystrophy (DMD)
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