Current Studies
Study 351-201
RecruitingA Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
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Indication
Duchenne Muscular Dystrophy
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Investigational Therapeutic
BMN 351 (Antisense oligonucleotide)
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Study Type
Phase 1/2
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Goal
This First-in-Human, open-label study is to evaluate the safety and tolerability of BMN 351 in children
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Status
This study is currently recruiting participants