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

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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Bibliographische Detailangaben
Veröffentlicht in:	JAMA network open 2021-01, Vol.4 (1), p.e2031851-e2031851
Hauptverfasser:	Willcocks, Rebecca J, Forbes, Sean C, Walter, Glenn A, Sweeney, Lee, Rodino-Klapac, Louise R, Mendell, Jerry R, Vandenborne, Krista
Format:	Artikel
Sprache:	eng
Schlagworte:	Case-Control Studies Child Child, Preschool Dystrophin - genetics Gene therapy Genetic Therapy - methods Genetics and Genomics Humans Magnetic Resonance Imaging - methods Muscle, Skeletal - diagnostic imaging Muscular Dystrophy, Duchenne - diagnostic imaging Muscular Dystrophy, Duchenne - therapy Online Only Research Letter Treatment Outcome
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Beschreibung
Zusammenfassung:	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.
ISSN:	2574-3805 2574-3805
DOI:	10.1001/jamanetworkopen.2020.31851