A phase I/IIa clinical trial of rAAVrh74.MCK.GALGT2 gene therapy in patients with Duchenne muscular dystrophy (DMD)

Trial Profile

A phase I/IIa clinical trial of rAAVrh74.MCK.GALGT2 gene therapy in patients with Duchenne muscular dystrophy (DMD)

Planning
Phase of Trial: Phase I/II

Latest Information Update: 09 Nov 2017

At a glance

  • Drugs RAAVrh74.MCK.GALGT2 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Pharmacogenomic; Therapeutic Use
  • Most Recent Events

    • 09 Nov 2017 New trial record
    • 03 Nov 2017 According to a Sarepta Therapeutics media release, the Investigational New Drug (IND) application for the GALGT2 gene therapy program has been cleared by the FDA and Nationwide Children's Hospital is on track to initiate this trial year-end 2017.
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