A phase I/IIa study of microdystrophin gene therapy for the treatment of Duchenne muscular dystrophy

Trial Profile

A phase I/IIa study of microdystrophin gene therapy for the treatment of Duchenne muscular dystrophy

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2017

At a glance

  • Drugs Microdystrophin gene therapy-Nationwide Childrens Hospital (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; Therapeutic Use
  • Most Recent Events

    • 06 Nov 2017 According to a Sarepta Therapeutics media release, this clinical trial will be conducted at Nationwide Childrens with Jerry Mendell, M.D. and Louise Rodino-Klapac, Ph.D. as the principal investigators.
    • 06 Nov 2017 According to a Sarepta Therapeutics media release, this trial is now open to enrollment and anticipated to be dosed at potentially therapeutic levels in mid-November 2017.
    • 06 Nov 2017 Status changed from planning to recruiting, according to a Sarepta Therapeutics media release.
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