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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

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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: 04 Mar 2026

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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

  • 23 Feb 2026 According to BioMarin Pharmaceutical media release, Phase 1/2 data presentation for 6 mg/kg and 9 mg/kg cohorts at Muscular Dystrophy Association (MDA) Clinical & Scientific Congress (March 8 to 11, 2026).
  • 23 Feb 2026 According to BioMarin Pharmaceutical media release, initial Phase 1/2 data demonstrating 5.0% mean absolute dystrophin expression (without double-correction for histologic adjustment for muscle content) at week 25 in the 9 mg/kg cohort. The 12 mg/kg dose cohort continues to enroll participants, with topline data readout from this cohort expected in 2H'26.
  • 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.

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