Trial Profile

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

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 23 Nov 2025

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At a glance

27 Oct 2025 According to BioMarin Pharmaceutical media release, the company expects to share a clinical update for the 6 mg/kg and 9 mg/kg cohorts by YE 2025.
04 Aug 2025 According to BioMarin Pharmaceutical media release, the company expects to share initial data by year-end.
19 Jun 2025 Planned End Date changed from 1 Dec 2025 to 30 Sep 2026.

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