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Congenital structural myopathies gene therapy - Myosana Therapeutics

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Drug Profile

Congenital structural myopathies gene therapy - Myosana Therapeutics

Latest Information Update: 27 Dec 2025

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At a glance

  • Originator Myosana Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Myotubularin replacements
  • 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

  • Research Congenital structural myopathies

Most Recent Events

  • 22 Sep 2025 Early research in Congenital structural myopathies in USA (Parenteral), before September 2025 (Myosana Therapeutics pipeline, September 2025)

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