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 Feb 2025

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At a glance

Drugs BMN 351 (Primary)
Indications Duchenne muscular dystrophy
Focus Adverse reactions; Proof of concept
Sponsors BioMarin Pharmaceutical

Most Recent Events

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).
05 Aug 2024 According to BioMarin Pharmaceutical media release, the company has completed enrollment of the first dose cohort, with data expected by year-end
20 Feb 2024 Status changed from planning to recruiting.

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