Duchenne Muscular Dystrophy

Current Studies

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Study 351-201

Recruiting

A 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

• Indication

  Duchenne Muscular Dystrophy

• Investigational Therapeutic

  BMN 351 (Antisense oligonucleotide)

• Study Type

  Phase 1/2

• Goal

  This First-in-Human, open-label study is to evaluate the safety and tolerability of BMN 351 in children

• Status

  This study is currently recruiting participants

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