Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy.

Willcocks, Rebecca J; Forbes, Sean C; Walter, Glenn A; Sweeney, Lee; Rodino-Klapac, Louise R; Mendell, Jerry R; Vandenborne, Krista · JAMA Netw Open · 2021

case_control · Level III

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Abstract

This case-control study uses magnetic resonance imaging and spectroscopy to evaluate the association between treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) and muscle quality in children with Duchenne muscular dystrophy.

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