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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne... This nonrandomized controlled trial analyzes safety, biological, and functional outcomes associated with the infusion of rAAVrh74.MHCK7.micro-dystrophin gene transfer in a small group of patients with Duchenne muscular dystrophy. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Neurology American Medical Association

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Publisher
American Medical Association
Copyright
Copyright 2020 Mendell JR et al. JAMA Neurology.
ISSN
2168-6149
eISSN
2168-6157
DOI
10.1001/jamaneurol.2020.1484
Publisher site
See Article on Publisher Site

Abstract

This nonrandomized controlled trial analyzes safety, biological, and functional outcomes associated with the infusion of rAAVrh74.MHCK7.micro-dystrophin gene transfer in a small group of patients with Duchenne muscular dystrophy.

Journal

JAMA NeurologyAmerican Medical Association

Published: Sep 15, 2020

References