AAVrh74.MHCK7.micro-Dystrophin - Nationwide Children's Hospital

Drug Profile

AAVrh74.MHCK7.micro-Dystrophin - Nationwide Children's Hospital

Alternative Names: AAVrh74.MHCK7.micro dystrophin

Latest Information Update: 07 Dec 2017

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Nationwide Children's Hospital; Sarepta Therapeutics
  • 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

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 06 Nov 2017 Phase-I/II clinical trials in Duchenne muscular dystrophy (In infants, In children) in USA (IV)
  • 06 Nov 2017 The US FDA approves IND application for AAVrh74.MHCK7.micro-Dystrophin in Duchenne muscular dystrophy
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