Research programme: Duchenne muscular dystrophy therapeutics - Genethon

Drug Profile

Research programme: Duchenne muscular dystrophy therapeutics - Genethon

Alternative Names: micro-dystrophin program - Genethon

Latest Information Update: 01 Aug 2017

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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 Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 27 Jul 2017 Pharmacodynamics data from a preclinical trial in Duchenne muscular dystrophy released by Sarepta Therapeutics
  • 21 Jun 2017 Genethon and Sarepta Therapeutics enter an option agreement for Duchenne muscular dystrophy therapeutics
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