Research programme: dysferlin gene therapies - Genethon

Drug Profile

Research programme: dysferlin gene therapies - Genethon

Alternative Names: AAV1 dysferlin gene therapy - Genethon; Adenoviral-associated vector-mediated dysferlin gene therapy - Genethon; Dystrophy-associated fer-1-like protein therapy - Genethon; Gene therapy for dysferlinopathies - Genethon; Girdle muscular dystrophy 2B gene therapy - Genethon; Miyoshi myopathy gene therapy - Genethon

Latest Information Update: 25 Feb 2016

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At a glance

  • Originator Genethon
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Muscular dystrophies

Most Recent Events

  • 25 Feb 2016 Preclinical development is ongoing in France
  • 17 Jul 2013 Preclinical development is ongoing in France
  • 01 Aug 2011 Preclinical development for Muscular dystrophies (dysferlinopathies) is ongoing in France
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