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Glycogen storage disease type III gene therapy - Genethon

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Glycogen storage disease type III gene therapy - Genethon

Latest Information Update: 04 Dec 2024

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At a glance

  • Originator Genethon
  • Class Gene therapies
  • Mechanism of Action Gene transference; Glycogen debranching enzyme system expression stimulants
  • 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

  • Clinical Phase Unknown Glycogen storage disease type III

Most Recent Events

  • 21 Oct 2024 Clinical trials in Glycogen-storage-disease-type-III (IV), before October 2024 (Genethon pipeline, October 2024)

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