A phase I/IIa study of microdystrophin gene therapy for the treatment of Duchenne muscular dystrophy
Phase of Trial: Phase I/II
Latest Information Update: 17 Jan 2018
At a glance
- Drugs Microdystrophin gene therapy-Nationwide Childrens Hospital (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions; Therapeutic Use
- 17 Jan 2018 According to a Parent Project Muscular Dystrophy media release, the first patient has been dosed with microdystrophin gene therapy by Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, and their team at Nationwide Children's Hospital. Preliminary results from this study are expected by mid-2018.
- 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.