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: 25 Jun 2025

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At a glance

19 Jun 2025 Planned End Date changed from 1 Dec 2025 to 30 Sep 2026.
19 Jun 2025 Planned primary completion date changed from 1 Dec 2025 to 30 Sep 2026.
19 Feb 2025 According to BioMarin Pharmaceutical media release, the company is initial proof-of-concept data at a scientific congress in the second half of 2025 (including muscle dystrophin levels after 25 weeks of dosing).

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