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Research programme: musculoskeletal disorder gene therapies - BioMarin Pharmaceutical

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Research programme: musculoskeletal disorder gene therapies - BioMarin Pharmaceutical

Latest Information Update: 23 Apr 2024

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At a glance

  • Originator BioMarin Pharmaceutical
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Musculoskeletal disorders

Most Recent Events

  • 23 Apr 2024 Preclinical trials in Musculoskeletal disorders in USA (Parenteral) before April 2024 (BioMarin Pharmaceutical pipeline, April 2024)
  • 22 Sep 2023 Early research in Musculoskeletal disorders in USA (Parenteral) (BioMarin Pharmaceutical pipeline, September 2023)

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