Duchenne Muscular Dystrophy Clinical Trials

Duchenne muscular dystrophy (DMD) is a recessive, X-linked neuromuscular disorder caused by a severe deficiency or the complete absence of dystrophin, a protein that helps protect muscle cells.

Learn more about our DMD clinical trials below.

BMN 351

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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

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Indication

Duchenne Muscular Dystrophy

Goal

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

Investigational therapeutic

BMN 351 (Antisense oligonucleotide)

Status

This study is currently recruiting participants

Study Type

Phase 1/2