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BMN 351

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Drug Profile

BMN 351

Alternative Names: BMN-351

Latest Information Update: 12 May 2026

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At a glance

  • Originator BioMarin Pharmaceutical
  • Class Antisense oligonucleotides
  • Mechanism of Action Dystrophin expression stimulants; Gene silencing
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 04 May 2026 Efficacy and pharmacodynamic data from a phase I/II trial in Duchenne muscular dystrophy released by BioMarin Pharmaceutical
  • 03 Apr 2026 Biomarin Pharmaceutical plans a phase II trial inDuchenne muscular dystrophy (In infants, In neonates, In children, In adolescents, In adults) in Italy, Spain, Netherlands, United Kingdom, Turkey (IV, Infusion) (EUCT2024-511656-41-00)
  • 24 Feb 2026 Interim pharmacodynamics data from a phase I/II trial in Duchenne muscular dystrophy released by BioMarin Pharmaceutical

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